On December 5, 2024, FDA published a new draft guidance on accelerated approval providing a much needed and substantial update to its guidance on the pathway. FDA’s application and use of accelerated approval has evolved dramatically since it was first developed by the Agency to …
The Winter Solstice, signifying the shortest day of the year, was Saturday, December 21. Although the earliest sunset of the year was actually in early December, this day also marked another premature sunset – the beginning of the end of the rare pediatric disease priority …
On July 17, 2024, FDA announced the establishment of a Rare Disease Innovation Hub (the “Hub”) to enhance and improve outcomes for patients with rare diseases and conditions by focusing on rare disease drug and biological product development across the Agency’s Center for Drug Evaluation …
Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program. The START pilot program was announced last September as a way to …
On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here). Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor developed by Italfarmaco, S.p.A., represents a new class of therapeutics to slow progression …
In 2009—15 years ago— the National Organization for Rare Disorders (NORD) announced the first U.S. recognition of Rare Disease Day. NORD’s announcement followed in the footsteps of European rare disease patient organization, Eurodis, who had celebrated the first Rare Disease Day the year before. Time flies when …
On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” As described by FDA’s press release, genetic metabolic diseases are conditions whereby a genetic mutation, generally one …
This time last year, we wrote about a long-overlooked FDA statutory authority and wondered if this provision, known colloquially as the “single study plus confirmatory evidence” pathway, was having a moment (previous post here). Two weeks ago, FDA published a draft of its latest drug …
In early 1984, a year after President Reagan signed the Orphan Drug Act into law, the FDA was not seeing the anticipated avalanche of requests for orphan drug designation. At that time, new Federal laws on drugs were far and few between. FDR signed the …
As we begin the final quarter of 2022 and the leaves here on the east coast begin to turn and fall, it seems the clock may be running out on FDA and the Center for Drug Evaluation and Research (CDER) to meet its goal of …
On September 29, 2022, FDA approved Amylyx’s NDA for its drug, Relyvrio (sodium phenylbutyrate/taurursodiol), for treatment of patients with amyotrophic lateral sclerosis (ALS) (see FDA announcement here). This approval decision charts a path for the exercise of appropriate flexibility in regulatory decisions for other rare …
On June 17, 2021, FDA gave notice of an upcoming public workshop focused on clinical trial designs for progressive multifocal leukoencephalopathy (PML). PML is a rare disease that occurs when the JC virus (JCV), generally thought to lie dormant in the adult population, takes advantage …
Yesterday’s FDA approval of Biogen’s Alzheimer’s drug, Aduhelm (aducanumab-avwa), is historic and is of a magnitude that it may be harbinger for future Agency actions, especially in neuropsychiatric conditions. This has been a decision we’ve been closely following. So much so, in November, right after …
On November 23, 2020, the FDA announced that it had approved Alnylam Pharmaceuticals’ Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria 1 (PH1), a rare metabolic disorder that causes recurrent kidney stones and loss of kidney function. In the Agency’s press release, it credited …
On November 13, 2019, the National Academies of Sciences, Engineering, and Medicine (NASEM) Forum on Regenerative Medicine hosted a workshop on Exploring Novel Clinical Trial Designs for Gene-Based Therapies. The Forum was tasked with exploring the design complexities and ethical issues associated with gene- and …
