Review Pathways
Meeting the regulatory gold standard for drug candidates in ultra-rare diseases can be impractical, a regulatory expert says, but greater collaboration and shared insights from regulatory reviews could help find a viable path forward.
The rate of non-submissions for combination therapies in England has been on a downward trend in the last two years.
Nipocalimab is an investigational FcRn blocker for treating generalized myasthenia gravis that was also recently filed for regulatory review in the US.
The number of standard and priority reviews also decreased significantly in FY 2023 compared to the previous year, which caused the fee for redeeming a voucher to rise.
A US FDA webinar for start-up sponsors showcased staff who sound enthusiastic about helping drug developers advance their projects, while also emphasizing the importance of early and continual engagement with the agency.
US FDA Office of New Drugs Director Peter Stein says review divisions have made the case that a discussion-only meeting would solicit the necessary input.
The European Medicines Agency is mulling how best to involve experts in its decision-making processes while avoiding potential conflicts of interest, says executive director Emer Cooke.
Sponsors Aim To Avoid Confirmatory Trial Hurdles That Plagued Ocaliva In Primary Biliary Cholangitis
The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion.
Advisory committee concerns cast clouds over Iterum’s oral antibiotic, Intercept’s Ocaliva, and perioperative immuno-oncology regimens, while CSL and Pfizer aim to take their hematology franchises in new directions.
Pink Sheet reporters and editors discuss Pfizer’s abrupt decision to withdraw Oxbryta, the US FDA’s Oncologic Drugs Advisory Committee bringing sponsors of approved products back to discuss labeling changes while competitors are pending, and the new CEO of a generic industry trade association.
Maturing data supporting first-line indications for Merck’s Keytruda, Bristol’s Opdivo and BeiGene’s Tevimbra show inadequate efficacy for patients at the lowest level of PD-L1 expression in esophageal and gastric cancers.
The program for collaborative gene therapy reviews with the European Medicines Agency is starting with simple applications, but after launch could be expanded to earlier discussions of CMC and pharm/tox data requirements, the FDA’s Nicole Verdun said.
The UK regulator approved 16 medicines containing a new active substance in the first half of this year, while the European Commission issued marketing authorizations for 20 NAS-containing drugs.
The Oncologic Drugs Advisory Committee will discuss restrictions for patients expressing PD-L1 in first-line labeling for Merck’s Keytruda, Bristol Myers Squibb’s Opdivo, and a pending NDA for BeiGene’s Tevimbra.
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
The refuse-to-approve decision for i02/Intarcia’s Type 2 diabetes combination product is the latest example of the FDA reaching final negative decisions on challenging regulatory cases.
Inflation accounted for a larger portion of user fee revenue target increases for fiscal year 2025, compared to previous years, according to a Pink Sheet analysis.
The European Medicines Agency has recommended the pan-EU approval of three rare disease drugs: Pfizer’s Hympavzi, Henlius Biotech’s Hetronify and ImmunoGen’s Elahere in addition to five other new medicines.
Reliance procedures have been used for most recent new drug approvals, and will continue to be employed in future, although the national approval route will play a key role in promoting innovation and protecting public health, says the UK regulator.
Obeticholic acid has not confirmed clinical benefit and the benefit-risk profile is not favorable in primary biliary cholangitis, the FDA advisory committee said. The agency must now decide whether to keep Ocaliva on the market with new study requirements or seek withdrawal.