The drug targets a rare form of ALS that is caused by a genetic mutation that leads to the accumulation of toxic levels of the SOD1 protein. The European Medicines Agency (EMA) estimates that this genetic mutation affects about 2% of people with ALS.
The approval, the first for patients with Friedreich's ataxia in the European Union, was based on a mid-stage study that showed patients who took Biogen's Skyclarys experienced an improvement in neurological functions such as speaking, swallowing and standing, compared to a placebo.
Biogen said it will turn its focus to other treatments for the fatal, mind-robbing disease. The company also is helping Japanese drugmaker Eisai sell another Alzheimer's treatment, Leqembi, which received full FDA approval last summer.
Leqembi is an antibody designed to remove sticky deposits of a protein called amyloid beta from the brains of Alzheimer's patients. It is the first treatment shown to slow progression of the disease for people in the earlier stages of Alzheimer's.
The drug, Zurzuvae, is expected to be commercially available for adults with PPD in December. The companies had sought U.S. approval for Zurzuvae to treat major depressive disorder (MDD), or clinical depression, and postpartum depression, but the Food and Drug Administration in August approved it only for PPD.
The Japanese drugmaker's review compared data for 72 patients with early Alzheimer's given Leqembi by subcutaneous injection to prior pivotal trial results from 898 patients who received the drug by infusion. The intravenous (IV) form of Leqembi won U.S. approval based on that larger 18-month study showing the drug, which works by removing sticky clumps of beta amyloid from the brain, slowed cognitive decline by 27 per cent for people with early Alzheimer's disease.
The development comes after a health ministry panel recommended the drug's approval in August, followed by standard approval from the U.S. Food and Drug Administration (FDA) in July. The drug is the first treatment shown to slow progression of the disease for people in the earlier stages of Alzheimer's.
Plano, Texas-based Reata focuses on developing treatments that regulate cellular metabolism and deal with inflammation in serious neurologic diseases. It makes Skyclarys, an FDA-approved treatment for the neurologic disorder Freidreich's ataxia.
Biogen said it would buy biotech firm Reata Pharmaceuticals for roughly $6.5 billion to expand its presence in the rare disease drugs market. Through the deal, Biogen will gain Plano, Texas-based Reata's recently approved drug Skyclarys to treat a rare genetic disorder that causes progressive damage to the nervous system.
Investors have pinned their hopes on Leqembi as the U.S drugmaker's money spinner multiple sclerosis and spinal muscular atrophy (SMA) treatments face fierce competition from cheaper versions and rival drugs.
Neurofilament protein is a marker of nerve cell degeneration. Higher levels of the protein are considered as a predictor of a more rapid decline in clinical function and greater risk of death in patients.
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