February is Rare Disease Month, an important time to celebrate successes in addressing the needs of our fellow 25 to 30 million Americans living with a rare disease, but also to renew our resolve in addressing the much larger remaining challenges – both new and …
February is Rare Disease Month, designated as such to raise awareness and to support the millions of individuals affected by rare diseases. It is also an extremely meaningful time for those of us at HPM who work regularly with sponsors to help get therapies to …
There are officially signs of life for FDA’s rare pediatric disease priority review voucher (RPD PRV) program! On September 17, the House Committee on Energy and Commerce marked up the Give Kids a Chance Act of 2025 (H.R. 1262), legislation that would reauthorize FDA’s RPD …
FDA’s recent approval of Papzimeos (zopapogene imadenovec-drba) highlights FDA’s use of regulatory flexibility in rare, serious diseases with unmet medical need. Approved on August 14, 2025, Papzimeos is the first treatment in the U.S. for recurrent respiratory papillomatosis (RRP), a rare disease characterized by recurrent …
There is much change happening at FDA. In the wake of the April 1st reduction in force, there has been a great deal of uncertainty about what the impacts will be. This rings particularly true amongst the rare disease patient community, along with many small …
We recently blogged about a new December 2024 draft guidance about accelerated approval (the “December 2024 draft guidance”). That post largely focused on endpoints as well as the broader context for when accelerated approval is appropriate. However, as we note in that post, the design, …
On December 5, 2024, FDA published a new draft guidance on accelerated approval providing a much needed and substantial update to its guidance on the pathway.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 …
We recently published the first part of our review of FDA’s draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” Questions and Answers Guidance (see previous coverage here). In this post we focus in on the draft guidance document’s chemistry, …
On November 19, 2024, FDA released a draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” As much of the content of this draft guidance for cellular and gene therapy (“CGT”) products is articulated elsewhere, this document serves as a …
The U.S. Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical …
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 …
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 …
On September 18, 2023, FDA published an updated, final iteration of guidance for immediate implementation entitled, “Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies.” This final guidance replaces and supersedes the March 2020 …
