rare-disease
4 stories tagged "rare-disease"
A $2.7 Million Gene Therapy, Approved Because Patients Needed Less Cornstarch
Ultragenyx's Genglycos won accelerated approval on a surrogate: a 31 percent cut in daily cornstarch. But the disease is low blood sugar, and by the company's own numbers the treated group spent slightly more time below 70 mg/dL, while liver enzymes spiked in 71 percent and every patient needed ster
InvestigationsOne Irish Plant Releases Both of Sanofi's Pompe Enzymes. In June, the FDA Called Its Drugs 'Adulterated.'
One Irish plant runs the final release for both of Sanofi's lifelong Pompe enzymes. It just failed an FDA inspection on that exact step, and now the company says the fix could take months while patients confirm their next shipments.
Drugs & FDAA $440,000 Drug Proved This Obesity Was Never About Willpower
Brain-tumor surgery can wreck the hypothalamus and leave hunger permanently switched on. Setmelanotide restored the missing signal in a real placebo-controlled trial, then arrived priced near half a million dollars a year.
Drugs & FDAFDA's New Gene Therapy Shortcut Leans on 'Platform Knowledge.' Six Months Ago, They Revoked Sarepta's After Three Patient Deaths.
FDA's new gene therapy draft guidance invites sponsors to lean on "platform knowledge" to streamline submissions, six months after the agency revoked Sarepta's AAVrh74 platform designation following three patient deaths.