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2 months ago | 
mondaq.com | Mark A. Tobolowsky |Charles Raver |James Valentine
We recently blogged about a new December 2024 draftguidance about accelerated approval (the "December 2024 draftguidance"). That post largely focused on endpoints as well asthe broader context for when accelerated approval is appropriate. However, as we note in that post, the design, timing of initiation,and timely conduct of confirmatory trials are also importantconsiderations in FDA's determination of whether acceleratedapproval is appropriate.
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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On December 5, 2024, FDA published a new draft guidance onaccelerated approval providing a much needed and substantial updateto its guidance on the pathway.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On December 5, 2024, FDA published a new draft guidance on accelerated approval providing a much needed and substantial update to its guidance on the pathway.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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Dec 9, 2024 | 
mondaq.com | Mark A. Tobolowsky |Charles Raver |James Valentine |Cheese Consumers
On November 19, 2024, FDA released a draft guidance titled "Frequently Asked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 As much of the content of this draft guidance for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 one-stop shop or "Cliffs
Notes" for the numerous guidance documents now covering CGT
product development.
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Dec 6, 2024 | 
thefdalawblog.com | Charles Raver |James Valentine |Mark A. Tobolowsky
On November 19, 2024, FDA released a draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” As much of the content of this draft guidance for cellular and gene therapy (“CGT”) products is articulated elsewhere, this document serves as a one-stop shop or “Cliffs Notes” for the numerous guidance documents now covering CGT product development.
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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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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.
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Oct 2, 2023 | 
thefdalawblog.com | James Valentine |Frank J. Sasinowski |Charles Raver
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.
