The rare pediatric disease priority review voucher program remains in its sunsetting state – the current authority only allows for granting of such vouchers if the drug was designated as a drug for a rare pediatric disease not later than December 20, 2024, and for …
We recently blogged about a new December 2024 draft guidance about accelerated approval (the “December 2024 draft guidance”). That post largely focused on endpoints as well as the broader context for when accelerated approval is appropriate. However, as we note in that post, the design, …
The Winter Solstice, signifying the shortest day of the year, was Saturday, December 21. Although the earliest sunset of the year was actually in early December, this day also marked another premature sunset – the beginning of the end of the rare pediatric disease priority …
On November 19, 2024, FDA released a draft guidance titled “Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products.” As much of the content of this draft guidance for cellular and gene therapy (“CGT”) products is articulated elsewhere, this document serves as a …
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 …
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 …
The Food and Drug Omnibus Reform Act (“FDORA”), enacted in December 2022, added a requirement that sponsors submit Diversity Action Plans (“DAPs”) for certain clinical studies involving drugs, biological products, or devices (codified at 21 U.S.C. § 355(z)(3) and 21 U.S.C. § 360j(g)(9)(A)). For drugs, …
The trio of CDER, CBER, and CDRH released a new draft guidance titled “Use of Data Monitoring Committees in Clinical Trials” that revises the 2006 guidance “Establishment and Operation of Clinical Trial Data Monitoring Committees” and, when final, will replace the 2006 guidance. The new …
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 …
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, …
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 …
On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022. Although fairly short and light on substance, it has the potential to …
Much has changed since the long-gone days of 2017. The Washington Nationals won the World Series, Presidential administrations have come and gone, and FDA has added new meeting types and formats to its menu. And so, FDA has issued a new draft guidance to bring …
CDER, CBER, and the Oncology Center of Excellence recently published a final guidance document titled “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products” as another part of its real-world evidence (“RWE”) Program. This finalized …
In January 2023, Vanda Pharmaceuticals, Inc. (Vanda) submitted two interesting and substantially similar citizen petitions (Docket Nos. FDA-2023-P-0313 and FDA-2023-P-0344) regarding its product Hetlioz (tasimelteon). Vanda requested that FDA revoke the approval of Apotex’s and Teva’s generic versions of Hetlioz on the grounds that the …
