Rare diseases
Blueprint has a next-generation systemic mastocytosis treatment, called elenestinib, that Sanofi CEO Paul Hudson told analysts provides an “opportunity to grow through the ‘30s.”
Arguably the most notable of the FDA’s upcoming decisions is that regarding Gilead’s twice-yearly HIV prophylaxis lenacapavir.
Although the FDA has rejected Stealth’s new drug application for Barth syndrome candidate elamipretide, the agency identified a potential accelerated approval pathway. The company has pared down its staff to conserve resources to fund a potential resubmission.
InflaRx’s vilobelimab met the bar for futility in a Phase III trial for the rare skin disease pyoderma gangrenosum.
HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy.
Prothena has already discontinued the development of birtamimab, and anticipates further spending cuts, including workforce reduction.
Beginning this week in Chicago, the American Association for Cancer Research’s annual conference will feature presentations that could have far-reaching implications for breast and blood cancers and more.
China continues to be a source of innovation as Pfizer strikes biggest pact yet; HHS provides more info on Trump’s Most Favored Nation executive order; FDA Commissioner Marty Makary and CBER director Vinay Prasad reveal new COVID-19 vaccine strategy following Novavax approval; ODAC underway after chaotic planning; more.
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
Govorestat failed to meet its primary endpoint in a Phase II/III trial for a rare form of Charcot-Marie-Tooth disease, a few months after the FDA rebuffed the same drug in a similar indication.
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