NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued positive opinions on a precision medicine for a genetic form of amyotrophic lateral sclerosis (ALS) and on a label expansion for a precision medicine for cystic fibrosis.
Biogen on Friday said CHMP issued a positive recommendation for Qalsody (tofersen) as a treatment for patients with a rare form of ALS linked to a mutation in the SOD1 gene. Qalsody, an antisense oligonucleotide, binds to and helps degrade SOD1 mRNA to reduce the production of the SOD1 protein.
The committee specifically recommended marketing authorization under exceptional circumstances, a form of authorization granted when the committee determines that the benefit-risk assessment for a drug is positive, but that it's unlikely that the company will be able to obtain comprehensive data due to the rarity of a disease.
CHMP made its decision after reviewing biomarker and clinical data from a Phase III study in which patients on Qalsody had a 60 percent decrease in plasma neurofilament light (NfL), a blood-based biomarker associated with axonal injury and neurodegeneration, compared to a placebo group after 28 weeks. There were also trends in the study suggesting that Qalsody improved patients' physical abilities based on the ALS Functional Rating Scale-Revised.
If the European Commission (EC) authorizes the drug, Qalsody will be the first treatment in the EU to target a genetic cause of ALS, according to Cambridge, Massachusetts-based Biogen. Last year, Qalsody became the first treatment in the US for treating this subpopulation of ALS patients.
Separately, Boston-based Vertex Pharmaceuticals on Friday said it received a positive opinion from CHMP to expand the availability of its cystic fibrosis treatment Kalydeco (ivacaftor) to patients with certain mutations in the CFTR gene who are as young as 1 month old. Kalydeco is already approved for patients ages 4 months and older in the EU. Last year, the US Food and Drug Administration approved a label expansion for Kalydeco, making it accessible to children as young as 1 month old based on Phase III data showing the treatment was safe for younger children.
The EC will now review CHMP's opinions on Qalsody and Kalydeco and decide whether to make these treatments available to patients in Europe. Biogen is expecting the commission's decision on Qalsody in the second quarter of 2024.