FDA Policy/General

Open Letter to Trump Urges FDA Push on Rare Diseases

Rare disease advocates issued an open letter Monday urging president Trump and Congress to push FDA to accelerate approvals for treatments targeting fatal genetic disorders, arguing that regulatory delays are costing patients valuable time. The letter, signed by advocates representing families affected by Duchenne muscular dystrophy, Huntington’s disease and Sanfilippo syndrome, criticized the agency for what it described as slow reviews and shifting regulatory standards for rare disease therapies.

The appeal comes amid leadership upheaval at FDA following the 5/12 departure of former commissioner Marty Makary. The advocates said the transition presents an opportunity for new agency leadership to prioritize faster access to experimental and potentially life-extending treatments.

The group also referenced interim FDA commissioner Kyle Diamantas and vacancies atop the agency’s drug and biologics centers, including the recent departure of former CBER director Vinay Prasad.

In the letter, families shared personal stories about watching relatives deteriorate from progressive neurological and muscular diseases while awaiting treatment options. The advocates called on FDA to make greater use of accelerated approval pathways and invoked Trump’s signing of the federal Right to Try Act during his first term.

“Rare disease families do not have time for bureaucracy,” the letter stated. “Every month without treatment means more muscle loss, neurological decline, irreversible damage, and too often, more funerals.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome