FDA Policy/General

Ultragenyx Enters Agreement to Sell 2nd Priority Review Voucher

Ultragenyx Pharmaceutical says it has entered into an agreement to sell to an undisclosed buyer a Rare Pediatric Disease Priority Review Voucher (PRV) for $203 million. Ultragenyx won the PRV upon FDA approval of Fayuvi (rebisufligene etisparvovec-hopf), also known as UX111, the first-ever FDA-approved treatment for mucopolysaccharidosis Type IIIA (MPS IIIA, Sanfilippo syndrome Type A).

Earlier this month, Ultragenyx agreed to sell another PRV to an undisclosed buyer for $210 million. FDA awarded the voucher in August upon approval of Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, for use in adult and pediatric patients eight years and older with glycogen storage disease Type Ia.

Container Closure System Guidance Comments

Pharmaceutical Research and Manufacturers of America (PhRMA) says it appreciates FDA’s attention to container closure systems in light of the significant technological advances that have emerged in material science and drug delivery systems since the agency’s 1999 guidance on the same topic. In a comment letter on an

Gottlieb Urges FDA Regulatory Pathway for ‘Clinical AI’

Former FDA commissioner Scott Gottlieb is calling on Congress to establish a new regulatory pathway for clinical artificial intelligence models developed and used within hospitals, warning that the absence of consistent federal oversight could undermine patient safety and limit broader commercialization of clinical AI technologies. In an October 8 commentary

FDA Launches Rare Disease Framework Initiative

FDA says it is launching a Rare Disease Framework Initiative “to reimagine the development of drugs and biologics for rare diseases.” In a statement, which includes a link to a new white paper on the initiative, the program will “develop an optimized, fit-for-purpose framework that reflects the realities

Bioresearch Monitoring Issue at Lovelace Institute

A 4/7-4/18/2025 FDA Bioresearch Monitoring Program inspection at Lovelace Biomedical Research Institute in Albuquerque, NM, found objectionable conditions in the facility’s role in conducting two nonclinical laboratory studies. A 10/6 Warning Letter says the specific violation was: * failure of the quality assurance unit to

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Container Closure System Guidance Comments

Gottlieb Urges FDA Regulatory Pathway for ‘Clinical AI’

FDA Launches Rare Disease Framework Initiative

Bioresearch Monitoring Issue at Lovelace Institute