FDA Grants ‘Breakthrough’ Designation to Synageva Drug

 

SEBELIPASE ALFA

STRUCTURAL FORMULA
SGGKLTAVDP ETNMNVSEII SYWGFPSEEY LVETEDGYIL CLNRIPHGRK 50
NHSDKGPKPV VFLQHGLLAD SSNWVTNLAN SSLGFILADA GFDVWMGNSR 100
GNTWSRKHKT LSVSQDEFWA FSYDEMAKYD LPASINFILN KTGQEQVYYV 150
GHSQGTTIGF IAFSQIPELA KRIKMFFALG PVASVAFCTS PMAKLGRLPD 200
HLIKDLFGDK EFLPQSAFLK WLGTHVCTHV ILKELCGNLC FLLCGFNERN 250
LNMSRVDVYT THSPAGTSVQ NMLHWSQAVK FQKFQAFDWG SSAKNYFHYN 300
QSYPPTYNVK DMLVPTAVWS GGHDWLADVY DVNILLTQIT NLVFHESIPE 350
WEHLDFIWGL DAPWRLYNKI INLMRKYQ 378
Glycosylation sites (N)
Asn-15 Asn-80 Asn-140 Asn-252 Asn-300

http://www.ama-assn.org/resources/doc/usan/sebelipase-alfa.pdf  see cas  no , codes etc

FDA grants ‘breakthrough’ designation to Synageva drug

BOSTON, May 20, 2013 (BOSTON HERALD)–Synageva BioPharma Corp., a Lexington-based biopharmaceutical company that develops drugs for rare diseases, said today that the U.S. Food and Drug Administration has granted breakthrough therapy designation to a drug calledsebelipase alfa to treat early onset lysosomal acid lipase deficiency, also known as Wolman disease.

The designation was based on data generated from clinical trials with sebelipase alfa in patients with early onset LAL Deficiency, officials said.

LAL Deficiency is a rare autosomal recessive lysosomal storage disorder caused by a marked decrease in LAL enzyme activity. Early onset LAL Deficiency is the most rapidly progressive form of LAL Deficiency and is usually fatal within the first six months of life, the company said, adding that affected infants develop severe malabsorption, growth failure and liver failure.

According to the FDA, breakthrough therapy designation is intended to speed up the development and review of drugs for serious or life-threatening conditions.