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Feb 4, 2025 | 
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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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On November 19, 2024, FDA released a draft guidance titled "Frequently Asked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 As much of the content of this draft guidance for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On November 19, 2024, FDA released a draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” As much of the content of this draft guidance for cellular and gene therapy (“CGT”) products is articulated elsewhere, this document serves as a one-stop shop or “Cliffs Notes” for the numerous guidance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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Oct 30, 2024 | 
mondaq.com | Mark A. Tobolowsky
The neonatal period is a unique and complex period of rapid
growth and development throughout the body, thus creating unique
and complex challenges in medical product development for this
population. These changes may affect the safety of a product used
in this population in ways that are not reflected in studies of the
same product in other populations.
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Oct 28, 2024 | 
thefdalawblog.com | Mark A. Tobolowsky
The neonatal period is a unique and complex period of rapid growth and development throughout the body, thus creating unique and complex challenges in medical product development for this population. These changes may affect the safety of a product used in this population in ways that are not reflected in studies of the same product in other populations.
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Oct 13, 2024 | 
thefdalawblog.com | Mark A. Tobolowsky
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 granted for drugs for serious or life-threatening rare diseases where the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years.
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Mar 8, 2024 | 
mondaq.com | Mark A. Tobolowsky |Michelle Butler
On Friday, February 23, 2024, FDA announced its final decision
to withdraw the approval of Pepaxto (melphalan flufenamide), which
was approved in February 2021 in combination with dexamethasone for
the treatment of adult patients with relapsed or refractory
multiple myeloma (RRMM) who have received at least four prior lines
of therapy and whose disease is refractory to at least one
proteasome inhibitor, one immunomodulatory agent, and one
CD38-directed monoclonal antibody.
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Feb 28, 2024 | 
thefdalawblog.com | Michelle Butler |Mark A. Tobolowsky
On Friday, February 23, 2024, FDA announced its final decision to withdraw the approval of Pepaxto (melphalan flufenamide), which was approved in February 2021 in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.
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Jan 2, 2024 | 
mondaq.com | Mark A. Tobolowsky
FDA, like much of the rest of the world, has been adjusting to
the rapid changes in our world these last few years. For nearly
three years following the declaration of the COVID-19 health
emergency, there were no in-person meetings held. Then, in early
2023, CDER and CBER started a slow phase-in of in-person meetings
in a hybrid format, with some attendees present only virtually.
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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...
