Regulatory
Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.
The rejection of Atara Biotherapeutics’ Ebvallo in January was a “complete reversal” of the conesensus FDA reviewers had come to, according to a former agency employee, who said manufacturing problems were the only approvability barrier for the drug.
A trial of a MacroGenics’ drug temporarily paused enrollment after several safety events, including a fatality. The deceased patient had developed a severe case of neutropenia and concurrent septic shock.
Eli Lilly notches another win over Novo Nordisk, as Zepbound bests CagriSema in a head-to-head trial sponsored by Novo; The FDA kicked off Rare Disease Week, providing draft guidance on its new plausible mechanism pathway, while a bipartisan senate hearing on Thursday will focus on the authorization process for rare conditions; Another leadership change shakes up CDC; and Gilead acquires CAR T partner Arcellx for nearly $8 billion.
Regulators overseeing rare disease treatments need better tools to weigh competing risks in real time. Sarepta Therapeutics’ Elevidys is a prime example of why.
In August last year, the Health Department cut around $500 million in mRNA research funding, with Health Secretary Robert F. Kennedy Jr. saying the agency would instead divert the money “toward safer, broader vaccine platforms.”
The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
Ralph Abraham, a vocal vaccine skeptic who served at the agency for just three months, has stepped down due to “unforeseen family obligations,” according to the CDC.
Bysanti is based on iloperidone, an active metabolite of a compound that forms the core of Fanapt, another drug by Vanda Pharmaceuticals.
Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.
PRESS RELEASES