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Jan 15, 2025 | 
mondaq.com | Charles Raver |James Valentine |Ellis F. Unger |Frank J. Sasinowski
On December 5, 2024, FDA published a new draft guidance onaccelerated approval providing a much needed and substantial updateto its guidance on the pathway. FDA's application and use ofaccelerated approval has evolved dramatically since it was firstdeveloped by the Agency to help address the HIV/AIDS epidemic inthe late 1980s.
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Jan 12, 2025 | 
thefdalawblog.com | James Valentine |Ellis F. Unger |Frank J. Sasinowski |Charles Raver
On December 5, 2024, FDA published a new draft guidance on accelerated approval providing a much needed and substantial update to its guidance on the pathway.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 help address the HIV/AIDS epidemic in the late 1980s.
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Jul 17, 2024 | 
thefdalawblog.com | James Valentine |Frank J. Sasinowski |Sarah L Wicks
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 and Research (“CDER”) and Center for Biologics Evaluation and Research (“CBER”).
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Jun 12, 2024 | 
thefdalawblog.com | James Valentine |Frank J. Sasinowski |Sarah L Wicks
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.
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Mar 26, 2024 | 
thefdalawblog.com | James Valentine |Frank J. Sasinowski |Charles Raver
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 of DMD and adds significantly to the armamentarium available to treat this relentlessly progressive and devastating condition.
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Mar 18, 2024 | 
mondaq.com | Frank J. Sasinowski
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 your goal is to support the thirty million Americans
with rare diseases.
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Mar 7, 2024 | 
thefdalawblog.com | Frank J. Sasinowski
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 your goal is to support the thirty million people that suffer from rare diseases.
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Dec 20, 2023 | 
mondaq.com | Mark A. Tobolowsky |James Valentine |Charles Raver |Frank J. Sasinowski
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 that leads to dysfunction of a key protein or enzyme, disrupts the chemical processes (metabolism) responsible for converting food into energy and eliminating metabolic...
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Dec 13, 2023 | 
thefdalawblog.com | Mark A. Tobolowsky |James Valentine |Charles Raver |Frank J. Sasinowski
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 that leads to dysfunction of a key protein or enzyme, disrupts the chemical processes (metabolism) responsible for converting food into energy and eliminating metabolic...
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Oct 27, 2023 | 
mondaq.com | James Valentine |Charles Raver |Frank J. Sasinowski
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 ). Two weeks ago, FDA published a draft of its latest drug
development guidance explaining how drug and biological product
developers can use this pathway to meet the statutory standard for
efficacy.
