A Novartis drug that offers a new way to treat a certain type of chronic myeloid leukemia now has the regulatory O.K. in Europe, a decision that comes less than a year after the small molecule secured a speedy approval from the FDA.
The European Commission approved the drug, asciminib, for patients with advanced cases of chronic myeloid leukemia, Novartis said on Monday. The drug will be marketed under the name Scemblix, the same name for the product in the U.S.
Chronic myeloid leukemia (CML) is a rare type of cancer that starts in the bone marrow cells responsible for forming blood cells. Standard CML treatment includes small molecule drugs that block tyrosine kinases, which are enzymes key to cancer growth. These tyrosine kinase inhibitors all work the same way, which poses a problem when the cancer develops resistance to the drugs. This class of medicines also comes with side effects that include nausea, muscle cramps and bone pain, fatigue, and rashes. While treatment with these drugs can lead to a life expectancy comparable to the general population, the side effects associated with the chronic therapy can cause patients to stop treatment.
Scemblix offers patients a drug alternative. While it is also a tyrosine kinase inhibitor, the small molecule is designed to target a different part of these enzymes, the ABL myristoyl pocket. Scientific literature describes this approach as “specifically targeting ABL myristoyl pocket” (STAMP). The regulatory nod in Europe makes Scemblix the first approved STAMP inhibitor for CML. The approval specifically covers Philadelphia chromosome-positive CML in the chronic phase, one of three phases of the disease.
The European regulatory decision for Scemblix is based on the results of a Phase 3 study that compared the Novartis pill to Bosulif, a tyrosine kinase inhibitor currently used to treat CML. The study enrolled 233 patients whose CML had been treated with at least two tyrosine kinase inhibitors.
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The main goal was measuring for a reduction in the BCR-ABL gene, which is found in CML cells, at 24 weeks. According to this measure, the reduction rate in Scemblix-treated patients was nearly double that of those who received Bosulif. Also, the discontinuation rate from adverse reactions was lower, 5.8% in the Scemblix arm compared to 21.1% in the Bosulif group. The results were confirmed in a follow-up with patients at 96 weeks. The most common side effects reported in clinical testing included muscle pain, upper respiratory tract infections, and a low platelet count. Data from the Phase 3 clinical trial were presented earlier this year during the annual meetings of the American Society for Clinical Oncology and the European Hematology Association.
The European Commission decision covers the use of Scemblix in patients previously treated with two or more tyrosine kinase inhibitors. The approval applies to all 27 European Union members, plus Iceland, Norway, and Liechtenstein.
“Approval of Scemblix from the European Commission is a critical milestone to help bring this novel treatment to patients living with CML in Europe,” Haseeb Ahmad, Novartis’s president, Europe Innovative Medicines, said in a prepared statement.
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