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`US010080788B2
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`(12) United States Patent
`US 10,080,788 B2
`(10) Patent No.:
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`(45) Date of Patent:
`*Sep. 25,2018
`Ruddy et al.
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`(56)
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`(58) Field of Classification Search
`A61K 38/00; A61K 38/1709; A61K 38/57;
`CPC
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`A61K 47/02; A61K 47/12; A61K 47/18;
`A61K 47/183; A61K 47/22; A61K 9/0019
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`See application file for complete search history.
`
`
`References Cited
`U.S. PATENT DOCUMENTS
`
`
`6,090,777 A
`7/2000 Hack et a1.
`
`
`
`
`
`
`
`6,875,432 B2
`4/2005 Liu et a1.
`
`
`
`
`
`
`
`
`7,067,713 B2
`6/2006 Nuijens et a1.
`
`
`
`
`
`
`
`7,544,853 B2
`6/2009 Nuijens
`
`
`
`
`
`
`
`7,837,992 B2
`11/2010 Gurewich et a1.
`
`
`
`
`
`
`
`
`7,897,561 B2
`3/2011 Kotwal et a1.
`
`
`
`
`
`
`8,071,532 B2
`12/2011 Mannesse et a1.
`
`
`
`
`
`
`
`
`RE43,691 E
`9/2012 Nuijens
`
`
`
`
`
`8,283,319 B2
`10/2012 Schulte et a1.
`
`
`
`
`
`
`
`
`8,415,288 B2
`4/2013 Mannesse et a1.
`
`
`
`
`
`
`
`
`8,501,705 B2
`8/2013 Christadoss et a1.
`
`
`
`
`
`
`
`8,652,477 B2
`2/2014 Schwaeble et a1.
`
`
`
`
`
`
`
`
`9,616,111 B2*
`4/2017 Ruddy ................... A61K 38/57
`
`
`
`
`
`
`
`
`9/2001 Schoenhofer et a1.
`2001/0019839 A1
`
`
`
`
`
`
`
`11/2002 Winkler et a1.
`2002/0168352 A1
`
`
`
`
`
`
`2005/0288218 A1
`12/2005 Davis et a1.
`
`
`
`
`
`
`
`2006/0142187 A1
`6/2006 Davis et a1.
`
`
`
`
`
`
`2006/0233776 A1
`10/2006 Heimburger et a1.
`
`
`
`
`
`
`2007/0093443 A1
`4/2007 Madison et a1.
`
`
`
`
`
`
`
`2007/0192882 A1
`8/2007 Dewald
`
`
`
`
`
`
`2010/0143325 A1
`6/20 10 Gurewich
`
`
`
`
`
`
`2012/0171206 A1
`7/2012 Tomlinson et a1.
`
`
`
`
`
`
`
`2012/0244139 A1
`9/2012 Madison et a1.
`
`
`
`
`
`
`
`2013/0244941 A1
`9/2013 Mannesse et a1.
`
`
`
`
`
`
`2014/0234293 A1
`8/2014 Basta et a1.
`
`
`
`
`
`
`
`2014/0242062 A1
`8/2014 Madison et a1.
`
`
`
`
`
`
`2014/0309175 A1
`10/2014 Zhao et a1.
`
`
`
`
`
`
`
`2014/0315826 A1
`10/2014 Zhao et a1.
`
`
`
`
`
`
`2014/0371425 A1
`12/2014 Kleinschnitz et a1.
`
`
`
`
`
`
`2015/0023977 A1
`1/2015 Fraunhofer et a1.
`
`
`
`
`
`
`FOREIGN PATENT DOCUMENTS
`
`
`2968434
`6/2017
`
`
`
`WO-1992/06203 A1
`4/1992
`
`
`
`
`
`WO-1992/22320 A1
`12/1992
`
`
`
`
`
`WO-1995/06479 A1
`3/1995
`
`
`
`
`
`WO-1997/22347 A1
`6/1997
`
`
`
`
`
`WO-2001/46219
`6/2001
`
`
`
`
`
`WO-2001/57079
`8/2001
`
`
`
`
`
`WO-2004/034971 A2
`4/2004
`
`
`
`
`
`WO-2004/110356 A2
`12/2004
`
`
`
`
`
`
`WO-2007/047995 A2
`4/2007
`
`
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`
`
`(Continued)
`
`
`OTHER PUBLICATIONS
`
`
`
`
`
`
`
`
`
`
`D’Agnillo F “Summary Basis for Regulator Acti0n7Cinryze”
`FDA. (Year: 2008).*
`
`
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`
`
`(54) C1-INH COMPOSITIONS AND METHODS
`FOR THE PREVENTION AND TREATMENT
`
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`OF DISORDERS ASSOCIATED WITH C1
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`ESTERASE INHIBITOR DEFICIENCY
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`(72)
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`( * ) Notice:
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`(71) Applicant: Shire ViroPharma Incorporated,
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`Lexington, MA (US)
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`Inventors: Stephen Ruddy, Exton, PA (US);
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`Mark Cornell Manning, Johnstown,
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`CO (US); Ryan Erik Holcomb, Fort
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`Collins, CO (US)
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`(73) Assignee: Shire ViroPharma Incorporated,
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`Lexington, MA (US)
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`Subject to any disclaimer, the term of this
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`patent is extended or adjusted under 35
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`U.S.C. 154(b) by 0 days.
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`This patent is subject to a terminal dis-
`claimer.
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`(21) Appl. No.: 15/837,677
`
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`
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`(22)
`Filed:
`Dec. 11, 2017
`
`
`
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`(65)
`Prior Publication Data
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`US 2018/0110843 A1
`Apr. 26, 2018
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`Related US. Application Data
`
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`(63) Continuation of application No. 15/411,744, filed on
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`Jan. 20, 2017, which is a continuation of application
`
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`
`
`No. 14/855,168, filed on Sep. 15, 2015, now Pat. No.
`
`
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`
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`9,616,111, which is a continuation of application No.
`PCT/US2014/030309, filed on Mar. 17, 2014.
`
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`
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`
`
`
`
`(51)
`
`
`
`
`
`
`
`
`
`
`
`(60) Provisional application No. 61/791,399, filed on Mar.
`15, 2013.
`
`
`
`Int. Cl.
`
`
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`(2006.01)
`A61K 38/00
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`(2006.01)
`A61P 7/00
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`(2006.01)
`A61K 38/55
`(2006.01)
`co 7K 14/81
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`(2006.01)
`A61K 38/57
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`(2006.01)
`A61K 9/00
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`(2006.01)
`A61K 47/02
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`(2006.01)
`A61K 47/12
`(2017.01)
`A61K 47/18
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`(2006.01)
`A61K 47/22
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`(2006.01)
`A61K 38/1 7
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`(2006.01)
`A61K 9/08
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`(2006.01)
`A61P 31/00
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`(52) us. Cl.
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`CPC ............ A61K 38/57 (2013.01); A61K 9/0019
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`(2013.01); A61K 9/08 (2013.01); A61K 38/00
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`(2013.01); A61K38/1709 (2013.01); A61K
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`47/02 (2013.01); A61K 47/12 (2013.01); A61K
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`47/18 (2013.01); A61K 47/183 (2013.01);
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`A61K 47/22(2013.01);A61P 31/00 (2018.01)
`Page 1 of 20
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`WO
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`WO
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`(Continued)
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`Primary Examiner 7 Christina Bradley
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`Assistant Examiner 7 Zachary J Miknis
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`(74) Attorney, Agent, or Firm 7 Proskauer Rose LLP;
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`Fangli Chen; Julio J. Mendez
`
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`ABSTRACT
`(57)
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`Compositions and methods for the treatment and/or preven-
`tion of disorders associated with C1 esterase inhibitor defi-
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`ciency are disclosed.
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`30 Claims, 2 Drawing Sheets
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`Specification includes a Sequence Listing.
`C SL EXHIBIT 1001
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`Page 1 of 20
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`CSL EXHIBIT 1001
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`US 10,080,788 B2
`Page 2
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`(56)
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`WO
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`WO
`
`WO
`
`WO
`
`WO
`
`WO
`
`WO
`
`WO
`
`WO
`
`WO
`
`
`
`
`References Cited
`FOREIGN PATENT DOCUMENTS
`
`
`
`WO-2007/073186 A2
`6/2007
`
`
`
`
`
`
`WO-2009/073569
`6/2009
`
`
`
`
`
`WO-2011/003098 A1
`1/2011
`
`
`
`
`
`
`WO-2011/107591 A1
`9/2011
`
`
`
`
`
`
`WO-2011/116291 A1
`9/2011
`
`
`
`
`
`
`WO-2013/013017 A2
`1/2013
`
`
`
`
`
`
`WO-2013/138694 A1
`9/2013
`
`
`
`
`
`
`WO-2013/138730 A1
`9/2013
`
`
`
`
`
`
`WO-2013/138731 A1
`9/2013
`
`
`
`
`
`WO-2014/160499 A2
`10/2014
`
`
`
`
`
`
`
`OTHER PUBLICATIONS
`
`
`
`
`
`
`
`
`
`
`Gower et al. “Hereditary Angioedema Caused by Cl-Esterase
`Inhibitor Deficiency: A Literature-Based Analysis and Clinical
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`Commentary on Prophylaxis Treatment Strategies” WAO Journal
`4:S9-S21. (Year: 2011).*
`
`
`
`
`Jiang et al. “Subcutaneous infusion of human C1 inhibitor in swine”
`
`
`
`
`
`
`Clinical Immunology 136:323-328. (Year: 2010).*
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`Opferkuch et al. “Clinical Aspects and Therapy of Hereditary
`Angioneurotic Edema” New Trends in Allergy. J. Ring et al. (eds).
`
`
`
`
`
`
`
`p. 272-278 (Year: 1981).*
`
`
`
`
`
`Anonymous. “Subcutaneous” The Pharmaceutics and Compound-
`
`
`
`
`
`
`ing Laboratory. http://pha1mlabs.unc.edu/labs/parenterals/subcumneous.
`
`
`
`
`
`
`
`
`
`htrn (Year: 2009).*
`
`
`
`Anonymous. “Cinryze” European Medicines Agency EMN175539-
`
`
`
`
`
`
`
`2013. (Year: 2013).*
`
`
`
`Aberer, W., Hereditary angioedema treatment options: The avail-
`
`
`
`
`
`
`
`ability of new therapies, Annals of Medicine, 44:523-529 (2012).
`
`
`
`
`
`
`
`
`
`
`Agostoni, A. et al., Hereditary and acquired angioedema: Problems
`
`
`
`
`
`
`and progress: Proceedings of the third Cl esterase inhibitors defi-
`
`
`
`
`
`
`
`
`
`ciency workshop and beyond, Journal of Allergy and Clinical
`
`
`
`
`
`
`
`
`
`Immunology, 114(3): 851-8131 (2004).
`
`
`
`
`
`
`
`
`
`
`
`
`Anonymous. Wikipedia extract: https://en.wikipedia.org/wiki/C1-
`inhibitor.
`
`Anonymous, Subcutaneous, http://pharmlabs.unc.edu/labs/parenterals/
`
`
`
`
`
`
`
`
`subcutaneoushtm. Published Jun. 14, 2010.
`
`
`
`
`
`
`Anonymous, http://www.cslbehring.com/s1/cs/enco/1255923338532/
`
`
`
`
`
`
`
`news/1255928832614/prdetail.htm?tabSelections:1255923338570
`
`
`
`
`
`
`&currentPage:2 ; May 3, 2012.
`
`
`
`
`Anonymous, Phases of Clinical Trials, http://www.virginia.edu/vpr/
`
`
`
`
`
`
`
`
`
`irb/HSRidocs/CLINICALiTRIALsiPhases.pdf. Published Jun. 12,
`
`
`
`
`
`
`
`
`
`2012.
`
`Anonymous, News Release, Halozyme Therapeutics (2012). http://
`
`
`
`
`
`
`
`www.halozyme.com/investors/news-releases/news-release-details/
`
`
`
`
`
`
`
`
`
`2012/ViroPharma-and-halozyme-Therapeutic s-Announce-Initiation-
`
`
`
`
`
`
`
`of-Phase-2b-Dose-Ranging-Combination-Study-for-Subcutaneous-
`
`
`
`
`
`
`
`
`Administration-of-Cinryze-C1-esterase-inhibitor-human-with-
`
`
`
`
`
`
`hyaluronidase-rhuPH20/default.aspx.
`
`
`
`
`Anonymous, “ViroPharma and Holozyme Therapeutics Announce
`
`
`
`
`
`
`
`Initiation of Phase 2b Dose Ranging Combination Study for Sub-
`
`
`
`
`
`
`
`
`
`cumneous Administration of Cinryze® (C1 esterase inhibitor [human])
`
`
`
`
`
`
`with Hyaluronidase (rHuPH20)” Press release, published Dec. 19,
`
`
`
`
`
`
`
`
`2012.
`
`Anonymous, CinryzeTM rescribin information, Nov. 2012.
`
`
`
`
`
`
`Anonymous, Observations under Section 21 in relation to the
`
`
`
`
`
`
`
`
`patentability of GB1519921.9 of Shire Viropharma Incorporated.
`
`
`
`
`
`
`
`Notice from the Observations mailed on Dec. 19, 2016 by the Great
`
`
`
`
`
`
`
`
`
`
`Britain Intellectual Property Oflice.
`
`
`
`
`Anonymous, Third Party Observations in relation to EP 14762343.
`
`
`
`
`
`
`
`3, acknowledged by European Patent Office on Jan. 19, 2017.
`
`
`
`
`
`
`
`
`
`Anonymous, Press Release by BMI Research on Mar. 5, 2012,
`
`
`
`
`
`
`
`
`
`“Subcutaneous Cinryze with rHuPH20 produced positive effects in
`
`
`
`
`
`
`
`
`prevention of HAO,” in connection with poster titled “Safety,
`
`
`
`
`
`
`
`
`
`
`Pharmacokinetics (PK), and Pharmacodynamics (PD) of Subcuta-
`
`
`
`
`
`neous (SC) Cinryze® (C1 Esterase Inhibitor [Human]) with Recom-
`
`
`
`
`
`
`
`binant Human Hyaluronidase (rHuPH20) in Subjects with Heredi-
`
`
`
`
`
`
`
`
`tary Angioedema (HAE).”
`
`
`
`
`Page 2 of 20
`
`
`
`
`
`
`Anonymous, CSL R&D Briefing, Dec. 5, 2012.
`Anonymous, ViroPharma Press release, ViroPharma Provides Update
`
`
`
`
`
`
`
`on Phase 2 Clinical Evaluation of Subcutaneous Cinryze® (C1
`
`
`
`
`
`
`
`esterase inhibitor [human]) with Recombinant Human Hyaluronidase
`
`
`
`
`
`
`
`(rHuPH20), Aug. 1, 2012.
`
`
`
`
`Anonymous, “Subcumneous” http://pha1mlabs.unc.edu/labs/parenterals/
`
`
`
`
`
`
`
`
`subcutaneoushtm. Published Jun. 14, 2010.
`
`
`
`
`
`
`Anonymous, “Plasma Derived Proteins and Enzymes” Bio Files
`
`
`
`
`
`
`
`
`2006, 1.5, 2. Sigma Aldrich. Published 2006.
`
`
`
`
`
`
`
`
`
`
`
`
`Anonymous, “C1 Esterase Inhibitor from human plasma” Sigma
`Aldrich E0518. Published 2006.
`
`
`
`
`Banerji, A., Current treatment of hereditary angioedema: An update
`
`
`
`
`
`
`
`on clinical studies, Allergy and Asthma Proceedings, 31(5):398-406
`
`
`
`
`
`
`
`
`
`
`(2010) (abstract only).
`
`
`
`
`Berinert® approval letter, FDA, Oct. 9, 2009, available at https://
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/
`ApprovedProducts/LicensedProductsBLAs/
`
`
`
`
`FractionatedPlasmaProducts/ucm186265.htrn (downloadedon May
`
`
`
`
`
`
`25, 2017).
`
`
`Berinert® Prescribing Information, CSL Behring, Jul. 2012.
`
`
`
`
`
`
`
`Berinert UK summary-product-characteristics Sep. 2015, available
`
`
`
`
`
`
`
`at http://www.medicines.org.uk/emc/print- document?documentId:
`
`
`
`
`
`
`
`
`
`21650 (downloaded on May 22, 2017).
`
`
`
`
`Bernstein, J.A., Hereditary angioedema: a current state-of-the-art
`
`
`
`
`
`review, VIII: current status of emerging therapies, Annals ofAllergy,
`
`
`
`
`
`
`
`
`Asthma & Immunology, 100:S41-S46 (2008).
`
`
`
`
`
`
`Bock, SC. et al., Human Cl Inhibitor: Primary Structure, cDNA
`
`
`
`
`
`
`
`
`Cloning, and Chromosomal Localization, 25 Biochem. 4292-301
`
`
`
`
`
`
`
`
`(1986). (“Bock”).
`
`
`
`Bork, K. et al., A Review of Hereditary Angioedema and Recom-
`
`
`
`
`
`
`
`
`
`binant Human Cl-Inhibitor Treatment, Hereditary Angioedema,
`
`
`
`
`
`
`
`European Respiratory Disease, 32-35 (2011).
`
`
`
`
`
`Bork, K. et al., Treatment of acute edema attacks in hereditary
`
`
`
`
`
`
`
`
`
`angioedema with a bradykinin receptor-2 antagonist (Icatibant), J
`
`
`
`
`
`
`Allergy Clin Immunol, 119(6):1497-1503 (2007).
`
`
`
`
`
`
`
`Bork, K., German Guideline for Hereditary Angioedema a due to
`
`
`
`
`
`
`
`Cl-INH Deficiency, German Association of Scientific Medical
`
`
`
`
`
`
`
`
`Societies, 1-24 (2011).
`
`
`Bowen, T. et al., 2010 International consensus algorithm for the
`
`
`
`
`
`
`
`
`
`diagnosis,
`therapy and management of hereditary angioedema,
`
`
`
`
`
`
`Allergy, Asthma & Clinical Immunology, 6(24):1-13 (2010).
`
`
`
`
`
`Bowen, T., Hereditary angioedema: beyond international consensusi
`
`
`
`
`
`
`circa Dec. 20107The Canadian Society of Allergy and Clinical
`
`
`
`
`
`
`
`
`
`Immunology Dr. David McCourtie Lecture, Allergy, Asthma &
`
`
`
`
`
`
`
`Clinical Immunology, 7(1):1-14 (2011).
`
`
`
`Brackertz, D. & Kueppers, F., Hereditary angioneurotic oedema,
`
`
`
`
`
`302(7830) Lancet 680 (1973).
`
`
`
`
`
`Bygum, A., Hereditary AngioedemaiConsequences of a New
`
`
`
`
`
`
`
`Treatment Paradigm in Denmark, Acta Derm Venerol, 94:436-441
`
`
`
`
`
`
`
`
`
`
`(2014).
`
`
`Caballero, T. et al., Consensus Statement on the Diagnosis, Man-
`
`
`
`
`
`
`
`
`agement, and Treatment of Angioedema Mediated by Bradykinin.
`
`
`
`
`
`
`Part II. Treatment, Follow-up, and Special Situations, J Investig
`
`
`
`
`
`
`
`Allergol Clin Immunol, 21(6):422-441 (2011).
`
`
`
`
`
`
`Castellano, G. et al., Endothelial-To-Mesenchymal Transition in
`
`
`
`
`
`
`
`Swine Renal Ischemia Reperfusion Injury is Mediated by Comple-
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`ment and AKT Pathway, Nephrology Dialysis Transplantation,
`27(2):ii1-ii2 (2012).
`
`
`
`
`
`
`
`
`
`
`
`
`Caspi et al. (2010). P&T, vol. 35, Issue 7, Jul. 2010, Product
`Profiler: BerinertTM.
`
`
`Castellano, G. et al., Therapeutic Targeting of Classical and Lectin
`
`
`
`
`
`
`
`
`Pathways of Complement Protects from Ischemia-Reperfusion-
`
`
`
`
`
`
`
`Induced Renal Damage, The American Journal of Pathology,
`
`
`
`
`
`
`
`
`176(4):1648-1659 (2010).
`
`
`
`
`Choi, G. et al., Recombinant human Cl-inhibitor in the treatment of
`
`
`
`
`
`
`acture angioedema attacks, Transfusion Practice, 47:1028-1032
`
`
`
`
`
`
`
`
`(2007).
`
`
`Cicardi, M. et al., Evidence-based recommendations for the thera-
`
`
`
`
`
`
`
`
`
`peutic management of angioedema owing to hereditary C1 inhibitor
`
`
`
`
`
`
`
`deficiency: consensus report of an International Working Group,
`
`
`
`
`
`
`
`
`European Journal of Allergy and Clinical Immunology, 67: 147-157
`
`
`
`
`
`
`
`
`(201 1).
`
`
`
`
`
`Page 2 of 20
`
`

`

`
`US 10,080,788 B2
`
`
`Page 3
`
`
`
`
`
`
`
`
`(56)
`
`
`
`References Cited
`OTHER PUBLICATIONS
`
`
`
`
`
`
`
`
`
`
`Cinryze® approval letter, FDA, Oct. 10, 2008, available at https://
`www.fda.gov/biologicsbloodvaccines/bloodbloodproducts/
`
`
`
`
`
`
`
`
`approvedproducts/licensedproductsblas/fractionatedplasmaproducts/
`ucm093602.htm (downloaded on May 25, 2017).
`
`
`
`
`
`
`
`
`
`
`
`
`
`“Cinryze (Cl-esterase Inhibitor) Developed by Lev Approved to
`Launch in the US”, Progress in Pharmaceutical Sciences, 33 (4),
`
`
`
`
`
`Dec. 31, 2009.
`
`
`Cinryze® FDA Briefing Document, Blood Products Advisory Com-
`
`
`
`
`
`
`
`
`mittee Meeting, May 1-2, 2008, available at https://www.fda.gov/
`
`
`
`
`
`
`
`
`
`
`ohrms/dockets/ac/O 8/briefing/2008-435 5B2-1b.htm (downloaded on
`
`
`
`
`
`
`
`May 23, 2017).
`
`
`Cinryze® Prescribing Information, ViroPharma Incorporated, Nov.
`
`
`
`
`
`
`
`2012. (“Cinryze® label”).
`
`
`
`ClinicalTrials.gov Identifier: NCT01912456, A Study to Evaluate
`
`
`
`
`
`
`the Clinical Efficacy and Safety of Subcutaneously Administered
`
`
`
`
`
`
`
`
`Cl-esterase Inhibitor in the Prevention of Hereditary Angioedema,
`
`
`
`
`
`
`
`
`(received Jul. 29, 2013), available at https://clinicaltrials.gov/ct2/
`
`
`
`
`
`
`
`
`
`
`
`show/NCT01912456 (downloaded on May 25, 2017).
`
`
`
`
`
`ClinicalTrials.gov Identifier: NCT00748202, Berinert® P Study of
`
`
`
`
`
`
`Subcutaneous Versus Intravenous Administration (PASSION), (Sep.
`
`
`
`
`
`
`4, 2008), available at https://clinicaltrials.gov/ct2/show/
`
`
`
`
`
`
`
`
`
`NCT00748202 (downloaded on Feb. 19, 2016).
`
`
`
`
`
`ClinicalTrials.gov Identifier: NCT01756157, Subcutaneous Cinryze
`
`
`
`
`
`
`With Recombinant Human Hyaluronidase for Prevention ofAngioedema
`
`
`
`
`
`
`
`Attacks, (Jun. 29, 2012), available at https://clinicaltrials.gov/ct2/
`
`
`
`
`
`
`
`
`
`show/NCT01756157 (downloaded on May 25, 2017).
`
`
`
`
`
`Connolly, B. D. et al., Weak Interactions Govern the Viscosity of
`
`
`
`
`
`
`
`
`
`Concentrated Antibody Solutions: High-Throughput Analysis Using
`
`
`
`
`
`
`
`the Diffusion Interaction Parameter, 103 Biophys. J. 69-78 (Jul.
`
`
`
`
`
`
`
`
`2012).
`
`Craig, Curriculum Vitae of Dr. Timothy Craig, D.O.
`
`
`
`
`
`Craig, Declaration of Dr. Timothy Craig.
`
`
`
`
`
`Craig, T. et al., WAO Guideline for the Management of Hereditary
`
`
`
`
`
`
`
`
`Angioedema, WAO Journal, 182-199 (2012).
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`Craig, T. et al., When is prophylaxis for hereditary angioedema
`necessary?, Annals of Allergy, Asthma & Immunology, 102:366-
`
`
`
`
`
`
`
`372 (2009).
`
`
`Cruz, M.P., Conestat Alfa (Ruconest) First Recombinant C1 Esterase
`
`
`
`
`
`
`
`
`Inhibitor for the Treatment of Acute Attacks in Patients with
`
`
`
`
`
`
`
`
`
`Hereditary Angioedema, Pharmacy and Therapeutics, 40(2):109-
`
`
`
`
`
`
`
`111, 114 (2015).
`
`
`
`CSL Behring Gmbh and CSL Behring LLC, Petitioners v. Shire
`
`
`
`
`
`
`
`
`
`Viropharma Inc., Patent Owner, Petition for Inter Partes Review,
`
`
`
`
`
`
`
`
`
`US. Pat. No. 9,616,111, filed May 31, 2017, pp. 1-79.
`
`
`
`
`
`
`
`
`
`CSL Behring Press Release dated May 3, 2012: “CSL Behring
`
`
`
`
`
`
`
`
`
`Initiates Study of Subcutaneous Administration of Cl-esterase
`
`
`
`
`
`
`
`
`Inhibitor in Patients With Hereditary Angioedema”.
`
`
`
`
`
`CSL Ltd. Half Year Update 2016-2017, signed by John Shine, Apr.
`
`
`
`
`
`
`
`
`
`
`
`2017, available at http://www.csl.com.au/docs/954/57/CSL7HYR177
`
`
`
`
`
`
`
`
`
`
`sec.pdf.
`
`
`D’Agnilo F. “Summary Basis for Regulatory Action%inryze”
`
`
`
`
`
`
`
`
`FDA. Published Oct. 9, 2008.
`
`
`
`
`Davis, A.E. III, New treatments addressing the pathophysiology of
`
`
`
`
`
`
`
`
`hereditary angioedema, Clinical and Molecular Allergy, 6(2):1-7
`
`
`
`
`
`
`(2008).
`
`
`Dec. 2012 Update to Clinical Trial NCT01756157, Subcutaneous
`
`
`
`
`
`
`
`Cinryze With Recombinant Human Hyaluronidase for Prevention of
`
`
`
`
`
`
`
`Angioedema Amtcks, (Dec. 24, 2012), available at https://clinicaltrials.
`
`
`
`
`
`
`
`gov/archive/NCT01756157/2012712724 (downloaded on May 25,
`
`
`
`
`
`
`
`2017).
`
`European Medicines Agency, Jun. 24, 2010, EMNCHMP/450053/
`
`
`
`
`
`
`
`
`2010, Evaluation of Medicines for Human Use, CHMP Assessment
`
`
`
`
`
`
`
`
`
`Report, Ruconest®, Procedure No. EMENH/C/001223, available
`
`
`
`
`
`
`at http://www.ema.europa.eu/docs/en7GB/documentilibrary/EPARi-i
`
`
`
`
`
`
`
`
`
`Publiciassessmentireport/human/OO1223/WC500098546.pdf.
`
`
`
`
`
`
`
`European Patent Application No. 147623433, Shire Reply dated
`
`
`
`
`
`
`
`
`Mar. 2, 2017.
`
`
`European Search Report for EP14762343.3, 9 pages (Jan. 28, 2016).
`
`
`
`
`
`
`
`
`Page 3 0f 20
`
`
`
`
`
`
`
`
`Excerpt from ClinicalTrialsgov archive (dated Apr. 11, 2012) for
`
`
`
`
`
`
`
`
`
`NCT01576523.
`
`Extract from “Ansel’s Pharmaceutical Dosage Forms and Drug
`
`
`
`
`
`
`
`
`
`Delivery Systems”; ninth edition; 2001; Chapter 5, pp. 162-170;
`
`
`
`
`
`
`
`
`
`Eds. Allen Jr. et al.
`
`
`
`
`Farkas, H. et al., Short-term prophylaxis in hereditary angioedema
`
`
`
`
`
`
`due to deficiency of the Cl-inhibitoria long-term survey, Allergy,
`
`
`
`
`
`
`
`67:1586-1593 (2012).
`
`
`
`
`Farkas, H., Pediatric hereditary angioedema due to Cl-inhibitor
`
`
`
`
`
`
`
`deficiency, Allergy, Asthma & Clinical Immunology, 6(18):2-10
`
`
`
`
`
`(2010).
`
`
`Farrell, C. et al., Population pharmacokinetics of recombinant
`
`
`
`
`
`
`
`
`human C1 inhibitor in patients with hereditary angioedema, British
`
`
`
`
`
`
`
`Journal of Clinical Pharmacology, 76:897-907 (2013).
`
`
`
`
`
`
`Fay, A. and Abinun, M., Current management of hereditary angio-
`
`
`
`
`
`
`oedema (C’l esterase inhibitor deficiency), J Clin Pathol, 55:266-
`
`
`
`
`
`
`
`270 (2002).
`
`
`Feussner, et al. (2014). Biochemical comparison of four commer-
`
`
`
`
`
`
`
`
`
`cially available C1 esterase inhibitor concentrates for treatment of
`
`
`
`
`
`
`
`
`hereditary angioedema. Transfusion 54: 2566-2573. Oct. 2014.
`
`
`
`
`
`
`
`
`Firszt, R. & Frank, M. M., An Overview of Novel Therapies for
`
`
`
`
`
`
`
`
`Acute Hereditary Angioedema, 11(6) Am. J. Clin. Dermatol. 383-88
`
`
`
`
`
`
`
`(2010).
`
`
`Frank, M. M. Declaration of Michael M. Frank, excerpted from the
`
`
`
`
`
`
`
`prosecution history of US. Pat. No. 9,616,111.
`
`
`
`
`
`
`
`Frank, M.M., Recombinant and Plasma-Purified Human Cl Inhibi-
`
`
`
`
`
`
`
`tor for the Treatment of Hereditary Angioedema, WAO Journal,
`
`
`
`
`
`
`
`
`829-833 (2010).
`
`
`
`Gatlin, L. A. & Brister Gatlin, C. A., Formulation and Administra-
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`tion Techniques to Minimize Injection Pain and Tissue Damage
`Associated with Parenteral Products, Chapter 17 in Injectable Drug
`
`
`
`
`
`
`
`Development Techniques to Reduce Pain and Irritation 401-21
`
`
`
`
`
`
`
`(Pramod K. Gupta & Gayle A. Brazeau, eds., CRC Press 1999).
`
`
`
`
`
`
`
`
`
`(“Gatlin”).
`
`
`GenBank: CAA30314.
`
`
`Gesuete, R. et al., Recombinant C1 Inhibitor in brain ischemic
`
`
`
`
`
`
`
`injury, Annals of Neurology, 66(3):332-342 (2009).
`
`
`
`
`
`
`
`Ghannam, A. et al., C1 Inhibitor as a glycoprotein: The influence of
`
`
`
`
`
`
`
`polysaccharides on its function and autoantibody target, 71 Mol.
`
`
`
`
`
`
`
`
`Immunol. 161-65 (2016).
`
`
`
`Ghazi, A. and Grant, J.A., Hereditary angioedema: epidemiology,
`
`
`
`
`
`
`management, the role of icatibant, Biologics: Targets and Therapy,
`
`
`
`
`
`
`
`
`7:103-113 (2013).
`
`
`
`
`Giatlin et al., “Formulation and Administration Techniques to
`
`
`
`
`
`
`
`Minimize Injection Pain and Tissue Damage Associated with Parenteral
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`Products,” extract from “Injectable Drug Development. Techniques
`to Reduce Pain and irritation”; 1999; Chapter 17.
`
`
`
`
`
`
`
`
`Gomella, L. G. & Haist, S. A., Clinician’s Pocket Reference
`
`
`
`
`
`
`
`
`
`(McGraw-Hill Professional 9th ed. 2002).
`
`
`
`
`
`
`
`Gower, R.G. et al., Hereditary Angioedema cause by Cl-Esterase
`
`
`
`
`
`
`
`Inhibitor Deficiency: A Literature-Based Analysis and Clinical
`
`
`
`
`
`
`
`
`Commentary on Prophylaxis Treatment Strategies, World Allergy
`
`
`
`
`
`
`Organization Journal, 4(2): 89-821 (2011).
`
`
`
`
`
`Gurewich, V. et al., Recombinant human Cl-inhibitor prevents
`
`
`
`
`
`
`
`
`
`non-specific proteolysis by mutant pro-urokinase during optimal
`
`
`
`
`
`
`
`
`
`fibrinolysis, Thrombosis and haemostasis, 102(2):279-286 (2009)
`
`
`
`
`
`
`
`
`(abstract only).
`
`
`
`Hack, C.E. et al., Target levels of functional Cl-inhibitor in heredi-
`
`
`
`
`
`
`tary angioedema, Allergy, 67:123-130 (2012).
`
`
`
`
`
`
`Hack, E. et al., Immuno-Safety of Recombinant Human C1 Inhibi-
`
`
`
`
`
`
`
`tor in Patients With Hereditary Angioedema: An Integrated Analy-
`
`
`
`
`
`
`
`
`sis, World Allergy Organization, S45 (2012).
`
`
`
`
`
`
`Harrison, R.A., Human C1 Inhibitor: Improved Isolation and Pre-
`
`
`
`
`
`
`
`liminary Structural Characterization, 22 Biochemistry 5001-07 (1983).
`
`
`
`
`
`
`Hofstra, J.J. et al., Pharmacokinetics, clinical efficacy and safety of
`
`
`
`
`
`
`
`
`C1 inhibitor concentrate (Cl-esteraseremmer-N) for treatment of
`
`
`
`
`
`
`hereditary (and acquired) angioedema, Academic Medical Centre
`
`
`
`
`
`
`
`Amsterdam, Sanquin Division of Plasma Products, 43.
`
`
`
`
`
`
`
`Hofstra, J. J. et al., Pharmacokinetics, Clinical Efiicacy and Safety
`
`
`
`
`
`
`
`of Plasma-Derived Nanofiltered C1 Inhibitor Concentrate for Treat-
`
`
`
`
`
`
`
`
`ment of Hereditary and Acquired Angioedema, Blood. 112(11):
`
`
`
`
`
`
`
`694-694 (2008).
`
`
`
`
`Page 3 of 20
`
`

`

`
`
`
`
`US 10,080,788 B2
`Page 4
`
`
`
`(56)
`
`
`
`References Cited
`OTHER PUBLICATIONS
`
`
`
`
`
`
`
`
`
`
`Hollingsworth, C., Dyax Pushes Toward Front of Race for HAE
`Therapy, Bioworld Today, (Sep. 25, 2008) http://searchproquest.
`
`
`
`
`
`
`
`
`
`com/professional/docview/10790039467ac>.
`
`
`
`
`Hossler, P. et a1., Optimal and consistent protein glycosylation in
`
`
`
`
`
`
`
`
`
`mammalian cell culture, 19(9) Glycobiology 936-49 (2009).
`
`
`
`
`
`
`
`Informed Consent Form for Enrollment for Adults (dated Apr. 20,
`
`
`
`
`
`
`
`
`
`
`2012), for Study No. CSL83972001, corresponding to ClinicalTrials.
`
`
`
`
`
`
`
`
`gov (National Institutes of Health) identification No. NCT01576523.
`
`
`
`
`
`
`International Search Report for PCT/US2014/030309, 5 pages
`
`
`
`
`
`
`
`
`
`(dated Nov. 7, 2014).
`
`
`
`
`Jiang, H. et a1., Subcutaneous infusion of human C1 inhibitor in
`
`
`
`
`
`
`
`swine, Clinical Immunology, 136:323-328 (2010).
`
`
`
`
`
`
`
`Jiang, H. et a1., Subcutaneous (SQ) versus intravenous (IV) infusion
`
`
`
`
`
`of C1 Inhibitor (inh) on blood levels in swine, 6th C1 Inhibitor
`
`
`
`
`
`
`
`
`
`
`Deficiency Workshop 46 (May 2009).
`
`
`
`
`Johann Wolfgang Goethe University Hospitals, Berinert P Study of
`
`
`
`
`
`
`
`Subcutaneous Versus Intravenous Administration (PASSION),
`
`
`
`
`
`ClinicalTrials.goviNCT00748202, (2011).
`
`
`
`
`Jolles, S. et al., New Frontiers in Subcutaneous Immunoglobulin
`
`
`
`
`
`
`
`Treatment, 1(1) Biol Ther 1-15 (2011).
`
`
`
`
`Karadi, et a1., 5th C1 Inhibitor Deficiency Workshop, 1-80 (May
`
`
`
`
`
`
`
`
`
`31-Jun. 3, 2007).
`
`
`
`Kawalec, P. et a1., Administration of conestat alfa, human C1
`
`
`
`
`
`
`
`
`
`
`esterase inhibitor and icatibant in the treatment of acute angioedema
`
`
`
`
`
`
`
`
`attacks in adults with hereditary angioedema due to C1 esterase
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`inhibitor deficiency. Treatment comparison based on systematic
`review results, Pneumonol Alergol Pol, 8195-104 (2013) (abstract
`
`
`
`
`
`
`
`
`only).
`
`Koles, K. et al., Influence oflacmtion parameters on the N-glycosylation
`
`
`
`
`
`
`of recombinant human C1 inhibitor isolated from the milk of
`
`
`
`
`
`
`
`
`
`
`transgenic rabbits, Glycobiology, 14(11):979-986 (2004).
`
`
`
`
`
`
`
`Koles, K. et at, N- and O-glycans of recombinant human C1
`
`
`
`
`
`
`
`
`
`
`inhibitor expressed in the milk of transgenic rabbits, Glycobiology,
`
`
`
`
`
`
`
`
`14(1):51-64 (2004).
`
`
`Kreuz, J, Berinert P Study of Subcutaneous Versus Intravenous
`
`
`
`
`
`
`Administration (PASSION) ClinicalTrialsgov NCT00748202, pub-
`
`
`
`
`
`
`
`lished Sep. 4, 2008.
`
`
`
`Latypov, R. F. et a1., Elucidation of Acid-induced Unfolding and
`
`
`
`
`
`
`
`
`Aggregation of Human Immunoglobulin IgG1 and IgG2 Fe, 287 J.
`
`
`
`
`
`
`
`Biol. Chem. 1381-96 (2012).
`
`
`
`
`
`Le Bas-Bernardet, S. et a1., Xenotransplantation of Galactosyl-
`
`
`
`
`
`
`
`
`Transferase Knockout, CD55, CD59, CD39, and Fucosyl-
`
`
`
`
`
`
`
`Transferase Transgenic Pig Kidneys Into Baboons, Transplantation
`
`
`
`
`
`
`
`Proceedings, 43:3426-3430 (2011).
`
`
`
`
`Levi, M. et a1., Self-administration of C1-inhibitor concentrate in
`
`
`
`
`
`
`
`
`patients with hereditary or acquired angioedema caused by Cl-in-
`
`
`
`
`
`
`
`hibitor deficiency, 117 J. Allergy Clin. Immunol. 904-08 (2006).
`
`
`
`
`
`
`
`
`(“Levi”).
`
`
`Lev Pharmaceuticals, Inc, Clinical Pharmacology Review, Division
`
`
`
`
`
`
`
`of Hematology Office of Blood Review & Research, 1-8 (Dec. 4,
`
`
`
`
`
`
`
`2007).
`
`
`
`
`
`
`
`
`
`
`Li, H. H., Declaration of Dr. Huamin Henry Li (dated May 12,
`2015).
`
`Li, H. H., Self-administered C1 esterase inhibitor concentrates for
`
`
`
`
`
`
`
`the management of hereditary angioedema: usability and patient
`
`
`
`
`
`
`
`acceptance, 10 Patient Preference and Adherence 1727-37 (2016).
`
`
`
`
`
`
`
`
`Lis, H. & Sharon, N., Protein glycosylation Structural and func-
`
`
`
`
`
`
`
`tional aspects, 218 Eur. J. Biochem. 1-27 (1993).
`
`
`
`
`
`
`Longhurst, H. J. et a1., C1 Linhibitor concentrate home therapy for
`
`
`
`
`
`
`
`hereditary angioedema: a viable, effective treatment option, 147(1)
`
`
`
`
`
`
`
`Clin. Exp. Immunol. 11-7 (2007).
`
`
`
`
`
`Longhurst, H. J. et a1., HAE international home therapy consensus
`
`
`
`
`
`
`
`document, 6(22) Allergy Asthma Clin. Immunol. 1-7 (2010).
`
`
`
`
`
`
`Longhurst, H.
`J. et a1., Prevention of Hereditary Angioedema
`
`
`
`
`
`
`
`
`Attacks with a Subcutaneous C1 Inhibitor, 376(12) N. Engl. J. Med.
`
`
`
`
`
`
`
`1131-40 (2017).
`
`
`Longhurst, H., Rhucin, a recombinant C1 inhibitor for the treatment
`
`
`
`
`
`
`
`of hereditary angioedema and cerebral ischemia, Current Opinion in
`
`
`
`
`
`
`
`Investigational Drugs, 9(3):310-323 (2008).
`
`
`
`
`
`
`Page 4 of 20
`
`
`
`
`
`
`
`
`Lucca, J.J.D. et al., Effects of Cl Inhibitor on Tissue Damage in a
`
`
`
`
`
`
`
`
`Porcine Model of Controlled Hemorrhage, Shock, 38(1):82-91
`
`
`
`
`
`
`(2012).
`
`
`Lumry, W. et a1., Nanofiltered Cl-Esterase Inhibitor for the Actute
`
`
`
`
`
`
`
`
`
`
`VIanagement and Prevention of Hereditary Angioedema Attacks due
`
`
`
`
`
`
`to C1-Inhibitor Deficiency in Children, The Journal of Pediatrics,
`
`
`
`
`
`
`
`
`162(5):1017-1022 (2013).
`
`
`
`
`VIachinig, T. and Waldhauser, H., Declaration of Dr. Thomas
`
`
`
`
`
`
`
`
`VIachinig and Hanno Waldhauser.
`
`
`
`
`VIahmood,
`I, Clinical Pharmacology Review, Published Dec. 4,
`
`
`
`
`
`
`2007.
`
`VIartinez-Saguer et a1., Pharmacokinetic Berinert P Study of Sub-
`
`
`
`
`
`
`
`cutaneous Versus Intravenous Administration in Subjects with Mod-
`
`
`
`
`
`
`
`erate Hereditary AngioedemaiThe Passion Study, 127 Allergy
`
`
`
`
`
`
`
`
`Clin. Immunol. AB104 (Feb. 2011) (Abstract 389).
`
`
`
`
`
`
`
`VIartinez-Saguer,
`I. et a1., Pharmacokinetic Berinert P study of
`
`
`
`
`
`
`
`
`
`subcutaneous versus intravenous administration in subjects with
`
`
`
`
`
`
`
`moderate hereditary angioedemaithe passion study, Journal of
`
`
`
`
`
`
`
`
`Allergy and Clinical Immunology, 127: AB104 (2011).
`
`
`
`
`
`
`
`VIartinez-Saguer,
`I. et a1., Pharmacokinetics of plasma-derived
`
`
`
`
`
`
`
`
`
`Cl-esterase inhibitor after subcutaneous versus intravenous admin-
`
`
`
`
`
`
`
`
`istration in subjects with mild or moderate hereditary angioedema:
`
`
`
`
`
`
`
`
`
`the PASSION study, Transfusion, 54:1552-1561 (2014).
`
`
`
`
`
`
`
`
`VIaterial Safety Data Sheet (MSDS) for Berinert® with a date of
`
`
`
`
`
`
`
`
`
`issue of Sep. 23, 2009.
`
`
`
`VIaurer, M. and Magerl, M., Long-term prophylaxis of hereditary
`
`
`
`
`
`
`
`
`
`angioedema wit

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