Hyman, Phelps & McNamara, P.C. (HPM), the nation’s largest law firm dedicated exclusively to FDA and life sciences law, is pleased to announce that Gayatri Rao, M.D., J.D., M.B.E. has joined the firm as a Director. Dr. Rao brings a rare combination of experience spanning, nearly …
As regulatory frameworks for orphan medicinal products continue to evolve on both sides of the Atlantic, understanding the practical and strategic implications is increasingly important. On March 17th from 10:00 to 11:00 ET, Pinsent Masons and Hyman, Phelps & McNamara, P.C. are pleased to host a …
If Part 1 of our Rare Disease Month series highlighted The Good, and Part 2 addressed The Bad, Part 3 turns to The Ugly. And by “Ugly,’ we mean something entirely different – members of Hyman, Phelps & McNamara will be well represented at Rare …
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 …
As some of you know, tucked inside the Consolidated Appropriations Act of 2026, was some legislation that FDA has been pushing for quite some time. In addition to the incredibly important renewal of the Rare Pediatric Disease Priority Review Voucher program (blog on that forthcoming), …
The American Conference Institute’s (“ACI”) FDA Boot Camp returns March 25-26, 2026, at the New York City Bar, New York, NY. This foundational training brings together life sciences attorneys, in-house counsel, and compliance professionals to dissect FDA law, policy, and enforcement trends. What You’ll Gain: A clear …
Commissioner Makary’s pronouncement in April of a new pathway for rare disease therapies based on a plausible mechanism of action or biological plausibility generated enormous excitement. The Commissioner’s general statement found meaningful expression in twin announcements this past Wednesday: one by Dr. Tidmarsh of a …
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 …
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 …
The fight between Jazz, Avadel, and FDA over narcolepsy drug sodium oxybate has been a long and arduous one. Starting in 2022 with a patent certification requirement, both Jazz and Avadel have sued FDA over this drug, with Jazz most recently bringing suit challenging FDA’s …
Back in July, the United States Supreme Court turned the world of administrative law on its head, adding new layers of judicial oversight to what might have previously been thought of as fairly non-descript Federal agency functions. One of those cases was Loper Bright v. …
The Rare Pediatric Disease Priority Review Voucher program has had a bit of a tumultuous history in its 12 short years of existence. Designed to incentivize the development of drugs for pediatric rare diseases where such development may not otherwise have occurred, vouchers may be …
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 …
The Supreme Court’s recent decision in Loper Bright v. Raimondo has done away with Chevron deference to federal agencies’ interpretation of ambiguous statutes, including the FDA. The decision commands that federal judges must make their own decisions in suits against FDA, considering—but not deferring to—the …
