In a first for the bloc, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for a new type of stem cell transplant therapy for certain blood cancer patients.
The marketing endorsement was awarded to ExCellThera’s Zemcelpro and covers a recommended approval in adults with blood cancers that require an allogeneic hematopoietic stem cell transplantation (HSCT) following myeloablative conditioning for whom no other type of suitable donor cells is available.
Zemcelpro contains stem cells from a donor’s umbilical cord blood, some of which have been grown and multiplied, the European Medicines Agency (EMA) said in a press release on the decision. By boosting the number of cells, Zemcelpro makes the stem cells from a small cord blood unit more effective, the European regulator explained.
For several blood cancers—including leukemias, which are detected in the blood; lymphomas, which are found in the lymph nodes; and myelodysplastic syndrome and myelomas, which are diagnosed in the bone marrow—the only current curative treatment option is allogeneic HSCT.
This type of transplant involves using donated stem cells to replace the patient’s bone marrow cells to form new marrow that produces healthy blood cells.
The CHMP based its Zemcelpro recommendation on pooled analysis of two single-arm trials that included 25 patients. The studies found that 84% of patients who received Zemcelpro achieved neutrophil engraftment, when donor stem cells establish themselves in the recipient’s bone marrow and produce the white blood cells known as neutrophils within a median span of 20 days.
Some 10,000 new blood cancer cases requiring bone marrow transplant crop up in Europe each year, ExCellThera estimates. Many of these patients do not have access to suitable donor cells largely because of the unavailability of matched donors, the company explained.
ExCellThera said it expects to gain marketing authorization from the EMA in roughly two months' time. After winning an endorsement from the CHMP, prospective medicines must go before the European Commission for a final approval verdict.
ExCellThera also plans to file for approval of Zemcelpro in the U.S., U.K. and other countries.
Madrigal’s Rezdiffra among others gaining CHMP nods
The committee has also given a thumbs up to Madrigal’s Rezdiffra for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), a disease where fat cells accumulate in the liver causing chronic inflammation. If the drug gains marketing authorization, it would become the first treatment in the EU for the condition. Fifteen months ago, Rezdiffra became the first drug approved for MASH in the U.S.
Also making the grade this month was Teva blockbuster Austedo for patients with moderate-to-severe tardive dyskinesia. The FDA approved the treatment in the indication eight years ago. Tardive dyskinesia is an irreversible movement disorder characterized by repetitive and uncontrollable movements of the tongue, lips, face, trunk and extremities and can be caused by medications used to treat mental health or gastrointestinal conditions.
Meanwhile, Pfizer spinout SpringWorks Therapeutics has picked up a CHMP nod for Ogsiveo in adults with progressing desmoid tumors, which are marked by noncancerous soft tissue growths in the limbs and abdomen that often cause severe pain and disfigurement. The FDA approved Ogsiveo in the same indication in 2023.
Also gaining a positive recommendation from the CHMP was Partner Therapeutics’ Imreplys for the treatment of patients exposed to myelosuppressive doses of radiation with hematopoietic sub-syndrome of acute radiation syndrome. The medicine is known as Leukine in the U.S. where it was approved in 2018.
CHMP signs off on label expansions for 6 drugs
Aside from new approval recommendations, the CHMP has also endorsed labeled expansions for a half-dozen established medicines.
The committee gave a thumbs up to Ipsen’s Cabometyx as a second-line treatment for patients with unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumors. The FDA signed off Cabometyx in the indication three months ago. The treatment, which has been on the market since 2016, brought home sales of $1.8 billion last year.
Also winning CHMP recommendations were Johnson & Johnson blockbusters Darzalex, for smoldering multiple myeloma, and Imbruvica, for patients with previously treated mantle cell lymphoma who would be eligible for autologous stem cell transplant. AbbVie partners with J&J on Imbruvica.
The committee further signed off on expansions for GSK’s lupus treatment Benlysta, Bayer’s prostate cancer drug Nubeqa and Sanofi’s multiple myeloma medicine Sarclisa.
CHMP opens re-examination of Lilly’s Kisunla
Elsewhere, fifteen months after giving a thumbs down to Eli Lilly’s Alzheimer’s disease treatment Kisunla in March of last year, the CHMP will give the medication a second look. The regulator said that it re-opened its examination of Kisunla on June 2. The FDA signed off on Kisunla in July of last year, opening up competition with Eisai and Biogen’s Alzheimer's rival Leqembi.