Regulatory Oversight and Compliance

RoslinCT to Manufacture CRISPR-Based Gene Therapy Casgevy

December 14, 2023

RoslinCT will manufacture Vertex Pharmaceuticals’ CRISPR-based gene therapy, Casgevy, recently approved for treating sickle cell disease and β thalassemia.

FDA Announces Advisory Committee for Genetic Metabolic Disease Treatments

December 13, 2023

The new advisory committee will provide the agency with technical and scientific advice and recommendations on potential treatments for genetic metabolic diseases.

Sanofi to Terminate Rare Disease Drug Licensing Agreement with Maze Therapeutics in Wake of FTC’s Challenge

December 13, 2023

Sanofi has issued a statement expressing its disappointment with FTC’s decision to block an exclusive licensing agreement with Maze Therapeutics for a rare disease drug candidate.

FDA Releases Compounding Guidance

December 11, 2023

The agency released two guidance documents on the interim policy on compounding using bulk drug substances for both outsourcing and non-outsourcing facilities.

Renewing the UK Voluntary Pricing Scheme

December 02, 2023

What does the 2024 VPAS mean for pharma?

Signing Off from Washington

December 02, 2023

FDA and industry face unprecedented political and policy challenges.

New Medicines, Markets, and Manufacture: CRISPR for Sickle Cell Disease and β-thalassemia

November 30, 2023

Exagamglogene autotemcel seen as synechdoche.

Consent Decrees Entered Against Utah Supplement Manufacturer and Distributors

November 20, 2023

Consent decrees were issued against Evig LLC, of St. George, Utah, and Premium Production LLC, of St. George, Utah, for unapproved dietary supplements.

Consent Decree Entered Against Arizona Company

November 20, 2023

The US District Court for the District of Arizona entered into consent decree against Smart Women’s Choice and its CEO to stop the company from distributing unapproved contraceptive drugs.

FDA Approves First Recombinant Treatment for Rare Blood Clotting Disorder

November 14, 2023

Adzynma provides a replacement for low levels of the ADAMTS13 enzyme in patients with congenital thrombotic thrombocytopenic purpura.