Regulation

Aug. 12 Quick Takes: Approvals for DMD, acute pain; plus Priority Review for Fabry, hypercholesterolemia therapies and more

FDA approves a second targeted DMD therapyNS Pharma Inc.’s Viltepso viltolarsen received accelerated approval from FDA to treat Duchenne muscular dystrophy in patients amenable to exon 53 skipping. Vyondys golodirsen from Sarepta Therapeutics Inc. (NASDAQ:SRPT) was the...

After speedy approval on immunogenicity data, Russia readies Phase III study of COVID-19 vaccine ‘Sputnik’

The Ministry of Health of the Russian Federation has approved a COVID-19 vaccine under emergency rules based on immunogenicity rather than protection data.  While COVID-19 vaccine developers have been gathering safety and immune response data...

China’s updated reimbursement list likely to see COVID-19 therapies, more anti-PD-1s

Monday marks the final day the public can comment on a draft plan for China’s next National Drug Reimbursement List, which could be updated to include countermeasures for COVID-19-associated respiratory disease as well as more...

Guardant’s liquid biopsy approval paves the way for broader adoption of solid tumor profiling

FDA’s approval of a liquid biopsy test from Guardant that produces a comprehensive tumor mutational profile may trigger broader adoption of the tumor screening technology to better match patients to targeted therapies. FDA approved Guardant360...

GSK first across finish line in closely watched BCMA race

Despite toxicity concerns, FDA approved GSK’s BCMA-targeted antibody-drug conjugate Blenrep late Wednesday, making it the first in a new class of drugs for multiple myeloma. The accelerated approval for GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) didn’t come...

Regulators agree on endpoints for COVID-19 therapeutics trials

International regulators have agreed upon the best endpoints for use in clinical trials for COVID-19 therapeutics, with mortality the key endpoint for hospitalized patients, while testing in mild patients should focus on disease progression or...

FDA makes Gilead’s Tecartus, for mantle cell lymphoma, third U.S.-approved CAR T

Kite’s Tecartus has become the third CAR T cell therapy, after Kymriah and Yescarta, to earn FDA approval. On Friday, FDA granted accelerated approval to Tecartus brexucabtagene autoleucel from the Gilead Sciences Inc. (NASDAQ:GILD) unit...

Data Bytes: CHMP’s July recommendations

Among the 11 new medicines recommended for approval by EMA’s CHMP is the first antibody-drug conjugate, and most advanced BCMA-targeted agent, for multiple myeloma patients: GSK’s Blenrep belantamab mafodotin. FDA’s Oncologic Drugs Advisory Committee unanimously...

PDUFA VII to be shaped by COVID-19 experience

The COVID-19 experience, including the benefits of decentralized trials and virtual regulatory meetings, will shape negotiations over reauthorizing the Prescription Drug User Fee Act for fiscal years 2023-27. Every PDUFA reauthorization has been accompanied by...

First EUA for pooled COVID-19 test points to effect of prevalence on performance

FDA’s first authorization of a pooled COVID-19 test makes it clear this resource-saving approach is best reserved for populations where disease prevalence is low, highlighting the need to quickly adapt testing strategies in response to...

July 15 Quick Takes: Early access in U.K. for Alnylam RNAi; plus Verrica, CytoDyn, J&J and Roche

U.K. clears early access to lumasiran The U.K.’s Medicines and Healthcare products Regulatory Agency is making lumasiran from Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) available to patients with primary hyperoxaluria type I before its approval by the...

GSK maintains lead in BCMA race with FDA panel’s recommendation

If FDA follows a Tuesday recommendation by ODAC, GSK’s belantamab mafodotin would be the first BCMA-targeted therapy to gain approval. The unanimous vote by FDA’s Oncologic Drugs Advisory Committee in favor of the BCMA-targeting antibody-drug...

Data Bytes: FDA approvals continue at solid pace despite pandemic

FDA’s Center for Drug Evaluation and Research has approved more NMEs in 1H20 than in the first half of any of the previous five years, even as the COVID-19 pandemic disrupts agency site inspections, clinical...

Global regulators at odds on Phase III efficacy threshold for COVID-19 vaccines

Regulators are aligning their guidance on COVID-19 vaccine development through the the International Coalition of Medicines Regulatory Authorities, but they have yet to agree on the criteria for vaccine efficacy. A report released last Friday...

Plans to resume domestic inspections could keep FDA on track with PDUFA timelines

Despite the pandemic, FDA is on track for a banner year for new drug approvals, and its plan to gradually resume domestic inspections starting the week of July 20 could help keep it on track...