How will emerging SMA therapies address longstanding unmet needs, and where will remaining market opportunies lie? Key findings: Biogen/Ionis’s disease-modifying antisense oligonucleotide therapy...
Is this the herald of a new era for amyloidosis? On August 10, 2018, the FDA approved Alnylam’s Onpattro (patisiran) for the treatment of ATTR-FAP (polyneuropathy associated with amyloid...
The FDA recently approved Rigel Pharmaceuticals’ Tavalisse (fostamatinib) for adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment...
Why 2018 Will be a Breakthrough Year for These Rare Diseases With Rare Disease Day upon us, we highlight critical advances in drug development expected in 2018 In the last several years, disease-...
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The intrinsic nature of rare diseases puts the patient front and center of research, awareness, and clinical development. With Rare Disease Day upon us, we ask how can pharma companies and patients...
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On December 19, 2017, the FDA approved Spark Therapeutics’ gene therapy Luxturna for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophies, which include retinitis...
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Expectations for Spark Therapeutics’ Gene Therapy Luxturna in Retinitis Pigmentosa and Leber Congenital Amaurosis The FDA’s January 2018 regulatory decision deadline on the marketing...
A dramatic increase in the number of cystic fibrosis patients eligible for highly-priced disease-modifying therapies In July, Vertex Pharmaceutical announced positive topline Phase II data for two...
Interest, investment, and innovation in the rare disease space has accelerated in recent years and continues to grow—nearly half of all agents approved by the FDA last year had previously received...