CHMP Recommends Approval for Novel Blood Cancer Therapy, Endorses Multiple Drug Expansions

Europe's Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for ExCellThera's Zemcelpro, a groundbreaking stem cell transplant therapy for blood cancer patients. This decision marks a significant milestone in the treatment of hematological malignancies and heads a list of positive recommendations issued by the committee this month.

ExCellThera's Zemcelpro: A New Hope for Blood Cancer Patients

Zemcelpro, developed by ExCellThera, has received a positive opinion from the CHMP for use in adults with blood cancers requiring allogeneic hematopoietic stem cell transplantation (HSCT) after myeloablative conditioning, where no other suitable donor cells are available. This innovative therapy utilizes stem cells from umbilical cord blood, which are expanded to increase their effectiveness.

The recommendation is based on pooled analysis of two single-arm trials involving 25 patients. Results showed that 84% of patients achieved neutrophil engraftment within a median of 20 days. With an estimated 10,000 new blood cancer cases annually in Europe requiring bone marrow transplants, Zemcelpro could address a significant unmet need.

ExCellThera anticipates receiving full marketing authorization from the European Medicines Agency (EMA) within two months and plans to file for approval in the United States, United Kingdom, and other countries.

CHMP Endorses Multiple New Treatments and Label Expansions

The CHMP's latest round of recommendations includes several other noteworthy approvals and label expansions:

1. Madrigal's Rezdiffra received a positive opinion for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), potentially becoming the first approved treatment for this condition in the EU.

2. Teva's Austedo gained a recommendation for patients with moderate-to-severe tardive dyskinesia, eight years after its FDA approval for the same indication.

3. SpringWorks Therapeutics' Ogsiveo received a nod for adults with progressing desmoid tumors, following its FDA approval in 2023.

4. Partner Therapeutics' Imreplys was endorsed for treating patients exposed to myelosuppressive doses of radiation with hematopoietic sub-syndrome of acute radiation syndrome.

Additionally, the committee recommended label expansions for several established medicines:

• Ipsen's Cabometyx for second-line treatment of unresectable or metastatic neuroendocrine tumors
• Johnson & Johnson's Darzalex for smoldering multiple myeloma and Imbruvica for previously treated mantle cell lymphoma
• GSK's Benlysta, Bayer's Nubeqa, and Sanofi's Sarclisa also received recommendations for label expansions in their respective indications

Eli Lilly's Kisunla Under Re-examination

In a notable development, the CHMP has reopened its examination of Eli Lilly's Alzheimer's disease treatment Kisunla. This comes 15 months after initially rejecting the medication in March of the previous year. The FDA approved Kisunla in July last year, setting the stage for competition with Eisai and Biogen's Leqembi in the Alzheimer's treatment market.

As the pharmaceutical landscape continues to evolve, these CHMP recommendations signal potential advancements in treatment options across various therapeutic areas, from rare blood cancers to more common conditions like Alzheimer's disease and metabolic disorders.