Category: rare disease
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STAT+: In a first, a nonprofit wins FDA approval for rare disease gene therapy
STAT+: In a first, a nonprofit wins FDA approval for rare disease gene therapy The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug company, but an Italian charity. The authorization of Waskyra on Tuesday for Wiskott-Aldrich…
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STAT+: J&J’s two-drug combo blocked tumors in advanced multiple myeloma
STAT+: J&J’s two-drug combo blocked tumors in advanced multiple myeloma This is the web version of ASH in 30 Seconds, STAT’s report from the American Society of Hematology meeting. And on the last day of ASH, the sun shone once again. Thanks for joining us on this hematology-oncology journey. We hope you’ve enjoyed our coverage.…
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STAT+: Roche suffers setback as court allows generic version of its rare disease drug to be sold in India
STAT+: Roche suffers setback as court allows generic version of its rare disease drug to be sold in India In the latest tussle over access to medicines, an Indian court has upheld a ruling that found it would be in the public interest to allow a generic company to sell a lower-cost version of a…
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STAT+: FDA approves ultra-rare disease drug that became a symbol of uncertainty over regulatory standards
STAT+: FDA approves ultra-rare disease drug that became a symbol of uncertainty over regulatory standards After months of uncertainty, the Food and Drug Administration offered accelerated approval for an ultra-rare disease drug that had become a symbol of the fraught balancing act between upholding regulatory standards and accommodating desperate patients and their families. The agency…
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STAT+: Cogent Biosciences-Blueprint Medicines tiff turns spicy on eve of immune drug study readout
STAT+: Cogent Biosciences-Blueprint Medicines tiff turns spicy on eve of immune drug study readout This story is an excerpt from Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. Cogent Biosciences and Blueprint Medicines are direct competitors who hate each other. During a talk at the recent…
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STAT+: Sarepta reports second patient death after treatment with Duchenne gene therapy
STAT+: Sarepta reports second patient death after treatment with Duchenne gene therapy Sarepta Therapeutics said Sunday that it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who received the treatment. Sarepta disclosed the first patient death — a 16-year-old…
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STAT+: Day 1 at ASGCT: Missing scientists, a gene editing failure, and a gene editing first
STAT+: Day 1 at ASGCT: Missing scientists, a gene editing failure, and a gene editing first NEW ORLEANS — The mood at the annual meeting of the American Society of Cell and Gene Therapy isn’t amazing. The biotech market has been bad for years. The FDA and NIH are shrouded in uncertainty. But many researchers…
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STAT+: Vertex reports an earnings miss and pause on cystic fibrosis trial
STAT+: Vertex reports an earnings miss and pause on cystic fibrosis trial Vertex Pharmaceuticals on Monday reported disappointing first-quarter earnings impacted by weaker-than-expected sales of its drugs for cystic fibrosis, and little or no contributions from a gene therapy for sickle cell disease and a recently launched pain medicine. A “tolerability issue” caused a temporary…