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First Gene Editing Therapy Among Seven Drugs On Track For EU-Wide Approval

CHMP Confirms Non-Renewal Of Conditional Authorization For GSK’s Multiple Myeloma Drug

 
• By Vibha Sharma

The European Medicines Agency has recommended seven new drugs for EU-wide approval, and OKd two others under its EU-M4all procedure that is meant for high-priority human medicines intended for markets outside the EU.

Casgevy Is A Personalized One-Off Treatment • Source: Shutterstock

The first CRISPR/Cas9 gene-editing therapy, Casgevy (exagamglogene autotemcel), is on track for pan-EU marketing approval as a treatment for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TBT) after receiving a thumbs up at the December meeting of the European Medicines Agency’s human medicines committee, the CHMP.

Casgevy is Vertex Pharmaceuticals/CRISPR Therapeutics’ cell-based gene therapy medicinal product that uses the novel CRISPR/Cas9 technology to edit the patient's own blood stem cells

More from Approvals

New EU Filings

 
• By Neena Brizmohun

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
• By Sue Sutter

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
• By Sue Sutter

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
• By Sue Sutter

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

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Opioid Safety: US FDA Hears Mixed Messages On Adding Posmarketing Studies To Labeling

 
• By Sue Sutter

Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.

How Tumult At US FDA Could Impact Merger and Acquisition Strategy

 
• By Joseph Haas and Nielsen Hobbs

With review and development uncertainly increasing, "it’s going to take longer for certain targets … to progress to a place where they’ve been derisked enough that big pharma is ready to write a big check," said Andrew Goodman of Paul Hastings.

US FDA Asks Adcomm If Opioid Postmarketing Studies Are Relevant, Warrant Action

 
• By Sue Sutter

Two US FDA advisory committees will discuss results from epidemiological studies that attempted to quantify the prevalence, incidence and risk factors for misuse, abuse, overdose and death with opioid analgesics.