Safety

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

Hematologists’ concerns about hepatoxicity with hemophilia A gene therapies may limit use, but Phase III data for Pfizer’s candidate at ASH show a lower rate of ALT increases than seen with BioMarin’s Roctavian.

Vital discussions are ongoing over which medicines containing per- and polyfluoroalkyl substances should be exempt from forthcoming wide-reaching EU restrictions on these substances.

Canada’s shortage of children’s analgesic/antipyretic OTCs in 2022 as COVID-19 and seasonal flu virus combined to drive stockpiling by consumers. Some poison control centers and hospitals offered recommendations about modifying adult doses to pediatric use.

Questions from researchers and public health advocates during workshop to discuss FDA authority under PREA Act point to agency and OTC NDA sponsors having additional boxes to check on potential unmet needs in drugs indicated for children and specifically on clarity of labeling for consumers of low health literacy, narrowing indications to prevent confusion and education about OTC drug formulations for health care providers.

Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.

Requests for “enforcement actions are not within the scope of FDA’s citizen petition procedures,” CDER says, rejecting petition dosing device firm Parenteral Technologies submitted as it prepares for workshop on Pediatric Research Equity Act requirements for OTC NDA sponsors.

UK pharma also reaches agreement in principle, subject to DoJ approval, to pay $70m to resolve a whistleblower complaint filed by Valisure, the testing lab which in 2019 raised concerns about a potential link between the use of drugs containing ranitidine, a histamine-2 blocker, and cancer.

NDAs for additional OTC products containing acetaminophen and/or NSAIDs and indicated for use by children between 2 and less than 12 years old would trigger compliance by application sponsors with the act passed in 2003 to address lack of pediatric use information in drug labeling.

The FDA has placed zetomipzomib on clinical hold a year after Kezar decided to focus solely on the product.