The US FDA approved Day One’s pediatric brain cancer drug Ojemda, ImmunityBio’s bladder cancer immunotherapy Anktiva, an uncomplicated UTI claim for Utility Therapeutics’ Pivya, which has a long history in Europe, and Pfizer’s hemophilia B gene therapy Beqvez.

The European Medicines Agency believes Idorsia’s Jeraygo should be approved for use in the EU at two different doses rather than just the one approved by the US Food and Drug Administration last month.

Obecabtagene autoleucel, Autolus Therapeutics’s investigational treatment for relapsed or refractory B cell precursor acute lymphoblastic leukemia, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The CHMP, the European Medicines Agency’s human medicines committee, will this week decide whether a range of new medicines merit being approved.

Notable new drug approvals in South Korea last year included Pfizer’s Tukysa, Genentech’s Columvi and BeiGene’s Tevimbra, but the tally did not include any domestically-originated products. Alzheimer’s disease therapy lecanemab may be on the horizon this year.

The US FDA’s approval of Lumicell’s optical imaging agent Lumisight makes a dozen novel approvals in 2024 for the Center for Drug Evaluation and Research.

Scientific advice could help companies make up for the lack of involvement in scoping, but slots are in short supply.

The Pink Sheet highlights recent comments and insights from pharma officials and executives on key issues we are covering.

The second-line indication comes with safety labeling recently approved for BCMA-targeted CAR-T competitor Abecma, particularly around increased early mortality and secondary malignancies.

Labeling describes early death imbalance that delayed approval of Bristol Myers Squibb’s CAR-T therapy for more than three months after its user fee goal, but does not add to boxed warning; J&J’s Carvykti is due for imminent US FDA action on its own early-stage myeloma bid.

Seven novel agents approved in March include one accelerated approval, two rare pediatric disease priority review vouchers, two breakthrough therapy designations and one regenerative medicine advance therapy designation.

Astellas's first-in class CLDN18.2-targeting antibody receives its first approval worldwide, while crovalimab and a number of drugs for rare diseases also receive nods from regulators and are now awaiting reimbursement price-listing.

The European Medicines Agency believes Idorsia’s Jeraygo should be approved for use in the EU at two different doses rather than just the one approved by the US Food and Drug Administration last month.

Sandoz argued against paying higher rebates for two of its drugs via the Centers for Medicare and Medicaid Services’ Medicaid drug rebate program in a US court six years after the suggestion was first made.

Obecabtagene autoleucel, Autolus Therapeutics’s investigational treatment for relapsed or refractory B cell precursor acute lymphoblastic leukemia, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The CHMP, the European Medicines Agency’s human medicines committee, will this week decide whether a range of new medicines merit being approved.

New EU guidance outlines the process through which scientific committees, national competent authorities and other relevant decision-makers can request real-world evidence studies for regulatory purposes.

Pink Sheet reporter and editors discuss Richard Pazdur’s call for more diverse opinions in FDA advisory committee meeting open public hearings, agency questions about the stakeholders sponsors consult to execute clinical trial diversity plans, and the pending review of Stealth BioTherapeutics’ application for the Barth Syndrome candidate elamipretide.

The Pink Sheet highlights recent comments and insights from pharma officials and executives on key issues we are covering.

Oncology Center of Excellence Director Richard Pazdur says he would like to see more diverse views during the OPH session, not just individuals curated by the sponsor to present ‘a very positive picture’ of the drug. An upcoming ODAC meeting will have everyone in-person except the OPH speakers, public and press.

The Barth Syndrome candidate application will be reviewed by the FDA, but Stealth BioTherapeutics' CEO tells the Pink Sheet that the company still does not have clear guidance on how or whether it should gather additional clinical data.

Labeling describes early death imbalance that delayed approval of Bristol Myers Squibb’s CAR-T therapy for more than three months after its user fee goal, but does not add to boxed warning; J&J’s Carvykti is due for imminent US FDA action on its own early-stage myeloma bid.

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

Pink Sheet reporters and editor discuss the implications of Amylyx following through on its pledge to remove Relyvrio from the market after its trial failed, an FDA idea to bring potential biomarkers for promising allergy and asthma treatments to advisory committees, and Japan’s PMDA opening an office in Washington D.C.

The Centers for Disease Control and Prevention’s ACIP is not able to take on all vaccine-like preventative medicines and still has to grapple with how to handle therapeutic vaccines. 

The combined format is the new default for briefing advisory committee members, US FDA Oncology Center of Excellence Director Pazdur declared.

The US FDA’s Oncologic Drugs Advisory Committee unanimously agreed that accelerated approvals should be enabled by multiple myeloma trials using MRD as a surrogate endpoint.

Pink Sheet reporter and editors discuss Richard Pazdur’s call for more diverse opinions in FDA advisory committee meeting open public hearings, agency questions about the stakeholders sponsors consult to execute clinical trial diversity plans, and the pending review of Stealth BioTherapeutics’ application for the Barth Syndrome candidate elamipretide.

US FDA’s oncology advisory committee will discuss two meta-analyses supporting use of MRD as a surrogate endpoint to support accelerated approval in multiple myeloma trials.

As sponsors complain about difficult-to-implement ACIP recommendations hampering uptake, the government says developers share some of the blame, but adds that it is working to ensure recommendations can be operationalized. 

Oncology Center of Excellence Director Richard Pazdur says he would like to see more diverse views during the OPH session, not just individuals curated by the sponsor to present ‘a very positive picture’ of the drug. An upcoming ODAC meeting will have everyone in-person except the OPH speakers, public and press.

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

Pink Sheet reporters and editor discuss the implications of Amylyx following through on its pledge to remove Relyvrio from the market after its trial failed, an FDA idea to bring potential biomarkers for promising allergy and asthma treatments to advisory committees, and Japan’s PMDA opening an office in Washington D.C.

OCE Director Richard Pazdur is known for cultivating 'Pazdur moments' during advisory committees when he feels the conversation has gone off track. A recent webinar with ODAC members created a perfect opportunity to promote his agenda.

Expert panels may be used to help the FDA decide when and how a new biomarker could be leveraged for regulatory purposes. Participants at workshop on allergy and asthma biomarkers also discuss need for standardization of food allergy trials and new trial design approaches. 

Moderna touts enriched Phase II/III population and severe RSV data as CDC advisory committee re-evaluates shared clinical decision-making and prepares for expected June votes on Moderna’s mRNA-1345 and expanded adult use of GSK’s Arexvy.