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 …
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Frank J. Sasinowski
- Is Confirmatory Evidence Having a Moment?October 11th, 2022
- A Historic Day in Drug Development: FDA Approves Amylyx’s Drug to Treat ALS, Demonstrating FDA’s Application of Appropriate Flexibility in Rare DiseasesSeptember 30th, 2022
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 …
- FDA Disease Specific Workshops: Clinical Trial Designs for Progressive Multifocal LeukoencephalopathyJune 21st, 2021
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 …
- FDA’s Accelerated Approval of Biogen’s Aduhelm for Alzheimer’s: A Sign of Applying the Emergency Use Standard Beyond COVID?June 8th, 2021
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 …
- FDA Credits Recent Drug Approval to Patient Community Engagement; We Applaud the Agency for the Recognition and Its Legacy of PFDDDecember 3rd, 2020
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 …
- Novel Clinical Trial Designs for Gene Therapies: An Exploration of Challenges by the National Academies’ Forum on Regenerative MedicineNovember 22nd, 2019
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 …
- HP&M’s James Valentine Named a 2019 RARE Champion of Hope; Moderates Global Rare Disease Town Hall with FDA and EMAJuly 1st, 2019
Last week, Global Genes, a global rare disease patient advocacy organization, announced the 2019 RARE Champion of Hope Awardees, which included Hyman, Phelps & McNamara, P.C.’s very own, James Valentine. This is a great honor, bestowed upon “true champions for rare disease”, “people who inspire …
- A Historic Day in Drug Development: AveXis Gene Therapy for SMA ApprovedMay 27th, 2019
On May 24, 2019, FDA approved Avexis’s BLA for its gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), for treatment of spinal muscular atrophy (SMA). This represents the first approval of a systemically-administered somatic gene replacement therapy. (The first gene therapy approved by FDA was Luxturna in Dec. …
- Breaking Down FDA’s New Rare Disease Natural History Studies Guidance: Practical ConsiderationsMarch 29th, 2019
On March 25, 2019, FDA issued a draft guidance, “Rare Diseases: Natural History Studies for Drug Development,” to help inform the design and implementation of natural history studies that can be used to support the development of drugs and biological products for rare diseases (hereinafter …
- Celebrating the Orphan Drug Act’s 35th Anniversary: HP&M Attorneys Author Proposal for Building an FDA Rare Disease Center of Excellence in Advance of EveryLife Foundation Scientific WorkshopSeptember 12th, 2018
As we celebrate the 35th Anniversary of the Orphan Drug Act (see our 30th anniversary post here), periodic consideration of opportunities to reform and refine the approach to rare disease medical product regulation is warranted – similar to the review that occurred 10 years ago, which …
- A Spark Points the Way Forward on Gene TherapyOctober 13th, 2017
Today (October 12, 2017) FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee unanimously recommended approval of Spark Therapeutics, Inc.’s Biologics License Application (BLA 125610) for Voretigene Neparvovec (also called LUXTURNA), a gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65 …
- Regenerative Medicine Advanced Therapy: FDA’s Newest Expedited Program Evolves to Keep Pace with RecommendationsSeptember 27th, 2017
If you read our blog, you already know that the 21st Century Cures Act included a provision that created the Regenerative Advanced Therapy designation program, or “RAT” (see our previous coverage of the Act here). You will also already know that the Center for Biologics …
- Accelerating Accelerated Approval & Other Drug Development Signals from FDA’s Approval of 1st Therapy for ChagasSeptember 13th, 2017
On August 29, 2017, FDA approved Chemo Research’s NDA for benznidazole for treating children with Chagas disease and granted the sponsor a Tropical Disease Priority Review Voucher (PRV) (see FDA Press Release here). This is the first drug to treat Chagas disease, a life-threatening parasitic disease that affects …