FDA Ruffles Feathers by Delaying DMD Tx Review

WASHINGTON — Two drugs are competing for FDA approval as treatment for Duchenne muscular dystrophy (DMD), and the agency upset DMD advocates by delaying review of one of the drugs. On Tuesday, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee discussed the safety and efficacy of BioMarin’s drisapersen (Kyndrisa), which is thought to …

FDA Officials, Advisors Split Over New DMD Therapy

WASHINGTON — The FDA approved the first-ever drug for Duchenne muscular dystrophy (DMD), despite internal disagreements over the drug’s efficacy. Eteplirsen (Exondys 51), a product of Sarepta Therapeutics of Cambridge, Mass., won approval from the agency — despite a split vote from its own advisory committee — to treat DMD patients who have a “confirmed …

FDA Advisory Panel Splits on Second DMD Drug

To find out what has happened since this story was originally published on April 26, click here. Weighing hard data against patient testimonials, an FDA advisory panel voted 7-6 that a new Duchenne muscular dystrophy (DMD) failed to meet the minimum standards for accelerated approval on Monday. The Peripheral and Central Nervous System Drugs Advisory …

Eteplirsen Approval Seen as Marking New Direction at FDA

On April 26, an FDA advisory committee voted 7-6 that the exon-skipping drug eteplirsen for Duchenne muscular dystrophy (DMD) failed to meet the standards needed for accelerated approval. It was widely assumed that the FDA would tell the drug’s developer, Sarepta Therapeutics, to try again with better data. That, of course, did not happen. In …

House Subcommittee Presses Forward on 21st Century Cures

WASHINGTON — Roughly 95% of rare diseases have no FDA-approved treatment. Congress hopes that by equipping government agencies with better resources, it can accelerate innovation and bring cures to patients faster. The latest draft of the 21st Century Cures bill would grant $10 billion in mandatory funding to the National Institutes of Health beginning in …

What Else is in the 21st Century Cures Act?

WASHINGTON — When most people think about the 21st Century Cures Act, they think about curing cancer and Alzheimer’s disease, and curbing the nation’s opioid epidemic. But the nearly 1,000-page healthcare spending bill, which President Obama signed in mid-December, also aims to reform the nation’s fragmented mental health system, improve access to electronic health data, …

ACA Repeal: It Takes a Village of Congressional Committees

WASHINGTON — The takedown of Obamacare is at the top of the GOP-led Congress’ agenda, and health policy experts gave MedPage Today an account of how that process could play out. In addition to dismantling the Affordable Care Act (ACA), legislators must also make key decision on bills that will impact healthcare programs for children …