United States
The Avlayah accelerated approval for treatment of the neurological and peripheral manifestations of Hunter syndrome was based on a surrogate biomarker and may help alleviate concerns about FDA flexibility in reviewing rare disease products.
The US FDA’s anti-allergy therapy review divisions are eager to support less burdensome development pathways for potential food allergy treatments, agency officials indicated during a recent “expert panel.”
The FDA said chronic under-collection of GDUFA revenue could create budget problems going forward unless the system is changed, but industry does not want to endanger the generic sector with higher fees.
Experts from Orchard Therapeutics, the UK drug regulator and Fondazione Telethon encouraged companies developing advanced therapies to speak to regulators in all planned launch regions, as expectations can vary significantly.
ImmunityBio's Patrick Soon-Shiong made several misleading efficacy claims on a podcast and TV ad about the company's cancer treatment, the FDA said in a warning letter.
The threat of Chinese dominance in the prescription drug sector appears to be a strong, unifying theme for legislators heading into the US Food and Drug Administration user fee reauthorization cycle, but two very different sets of policy issues must be addressed.
Payroll costs in the prescription drug user fee program increased in fiscal year 2025 despite the reduction-in-force and voluntary departures, which could impact industry efforts to reduce the annual charges.
The US FDA aims to prevent poor quality or incomplete responses to Form 483 inspection observations with a new draft guidance that describes the structure and content for concise, factual and effective corrective action responses.
Sponsors thought a shorter goal date extension was appropriate if a facility received the unofficial potential Official Action Indicated tag, but the FDA said it needs the extra time to complete the final steps in the application review.
Ascent had hoped to join a growing list of US mirabegron competitors, but a Delaware court found its ANDA would infringe valid Astellas patents covering the sustained-release formulation.
While regulators are “catching up” to the use of artificial intelligence in clinical development, some skepticism remains around the use of AI to generate synthetic patient data, says Alexion’s Nick France.
The US FDA will seek industry feedback on the Commissioner’s National Priority Voucher pilot during a June public meeting as the ultra-fast drug review program marks its first year amid growing scrutiny of its scope, timelines, and sponsor obligations.
In a signal that prescription drug and generic drug user fee talks are nearing completion, the FDA and industry are preparing to start biosimilar user fee reauthorization talks with America First and other issues potentially looming.
Pink Sheet editors discuss the impact of a federal judge’s decision that the recent ACIP membership turnover and vaccine schedule changes likely violated statutes, as well as the FDA’s look at new opioid disposal requirements.
Lawyers from Sidley Austin tell the Pink Sheet how European drug manufacturers and marketing authorization holders should proactively deal with warning letters from the US Food and Drug Administration.
With the US CDC Advisory Committee on Immunization Practices sidelined by the courts, the Pink Sheet imagined how a past version of the panel may have spent the spring, including tracking a fast‑moving pipeline of flu, RSV, Lyme, dengue, and next‑generation combination vaccines.
The agency is conducting further investigation of reports of altered skin sensation that occurred more frequently with the 7.2 mg semaglutide dose than with lower doses of the GLP-1 inhibitor.
The agency offered to drop annual IND fees for sponsors not conducting Phase I trials in the US, but industry still raised several concerns about fee incentives for onshoring clinical development.
Artificial intelligence in pharmaceutical manufacturing is increasing in prevalence, offering a unique opportunity for global regulators to align on expectations, industry group IFPMA says.
A US lawmaker described program reform goals as returning the program to its original focus on supporting federally qualified health centers and rural hospitals.