| |   | Good morning, all. Damian here with a milestone in the field of gene therapy, a setback in Huntington's research, and the dramatic reveal of a bidding-war loser. | | | First hemophilia gene therapy wins European approval European regulators approved BioMarin’s one-time treatment for patients with hemophilia A, the company said yesterday, marking a long-awaited milestone for a promising medicine.
The treatment, called Roctavian, has conditional approval for people with severe hemophilia A, defined as patients who need regular administrations of prophylactic treatment to ward off dangerous bleeding episodes. In clinical trials, a single dose of Roctavian demonstrated a 95% reduction in bleeding episodes over four years and led to a 96% improvement in patients’ need for drugs that improve blood clotting.
BioMarin expects to file a marketing application with the FDA by the end of September, and analysts expect Roctavian to win U.S. approval by the middle of next year. | Novartis pauses a Huntington’s trial over safety concern Novartis has suspended dosing in a Phase 2 Huntington’s disease study after some patients showed signs of nerve damage, the company said yesterday.
The decision follows a safety review by the study’s independent monitors, Novartis said in a letter to the Huntington’s Disease Society of America, concluding that the drug, branaplam, might be causing peripheral neuropathy. The company said it plans to gather more data in the coming months before making a decision on next steps.
Branaplam, previously studied as a treatment for spinal muscular atrophy, is among the few potential medicines for Huntington’s in development. The disease has vexed the pharmaceutical industry for decades, as seemingly promising ideas have repeatedly failed to slow its degenerative effects in pivotal trials. Last year, Roche terminated a Phase 3 study of a closely watched Huntington’s therapy after concluding it was performing no better than placebo. | This fall, join STAT live in DC Learn from the researchers, patient advocates, and lawmakers driving the next breakthroughs in rare disease research this September 15. Stay once the conversation is complete for a networking and cocktail hour. Purchase your ticket now (or unlock a free pass as a STAT+ subscriber). | Do patients want an eye drug that hasn’t improved vision? That’s the question facing Apellis Pharmaceuticals as it nears an expected FDA approval for an ocular injection meant to treat a common cause of vision loss.
The news is that, according to two years of data disclosed yesterday, Apellis’ drug, pegcetacoplan, had a significant and sustained effect on the size of eye lesions for patients with a late-stage form of macular degeneration called geographic atrophy. However, that same study found that pegcetacoplan was no better than placebo at slowing patients’ loss of vision, even after 24 months of treatment.
Apellis has argued that two years is simply not enough time to observe pegcetacoplan’s potential to preserve vision, and analysts expect the drug’s effect on lesions to be enough to convince the FDA, which is slated to make an approval decision by Nov. 26. But whether prescribers and patients believe the data justify monthly needles in the eye remains to be seen. | J&J lost the Global Blood auction Earlier this week, Global Blood Therapeutics disclosed that there were two unnamed rival bidders in the run-up to Pfizer’s $5.4 billion acquisition of the company. Yesterday, Bloomberg reported that one of them was Johnson & Johnson.
J&J was the firm described as “Company A” in Global Blood’s requisite filing with the SEC, according to Bloomberg, which means the world’s largest health care company made a final bid of $65 a share before losing out to Pfizer’s deal-sealing offer of $68.50.
The revelation suggests that J&J, in its first year under new CEO Joaquin Duato, is taking an aggressive approach to dealmaking. According to Global Blood’s filing, the company wasn’t for sale until May, when a representative from the pharmaceutical giant formerly known as Company A reached out to CEO Ted Love and made a $55-a-share offer. | More reads - Inovio agrees to shell out $44 million to settle Covid-19 vaccine lawsuit. Endpoints
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| Thanks for reading! Until tomorrow,  | | | |
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