Genentech Gets FDA OK for Injectable Rare Neuroimmune Disease Drug

Xconomy San Francisco — 

A drug that can be administered at home to treat a rare central nervous system disorder that damages the optic nerves and spinal cord now has FDA approval, marking the third nod the agency has given to a therapy for the condition.

The drug, satralizumab (Enspryng), from Roche’s South San Francisco-based subsidiary Genentech, is for adults with neuromyelitis optica spectrum disorder (NMOSD) who have antibodies against aquaporin-4 (AQP4). The antibodies are believed to play a role in the immune overreaction that characterizes the rare disease. Sometimes misdiagnosed as multiple sclerosis, NMOSD can lead to blindness, muscle weakness, and paralysis.

Designed to target activity by the protein IL-6 that plays a role in the inflammation associated with NMOSD, satralizumab—created by Japan’s Chugai, part of Roche—is the only approved treatment for the condition that can be delivered subcutaneously.

Genentech says the drug uses “recycling” antibody technology to extend the duration it can circulate—the same technology that underpins ocrelizumab (Ocrevus), a multiple sclerosis drug the FDA OK’d in 2017 for the most severe cases of the disease. This technology permits dosing every four weeks, following an initial dose.

Approval for the new Genentech drug was based on results from two Phase 3 clinical trials in which treatment with the drug alone and in combination with immunosuppressant therapy was compared to a placebo. The goal of the trials was to reduce the risk of relapse, which, at 96 weeks, the drug achieved. Common adverse reactions were colds, headaches, upper respiratory tract infections, stomach inflammation, rash, joint pain, extremity pain, fatigue, and nausea.

About 200,000 people globally are affected by the condition, which is most common among women in their 30s and 40s and among people of African or Asian background.

The most recently approved treatment for the disorder is Gaithersburg, MD-based Viela Bio (NASDAQ: VIE) drug inebilizumab (Uplizna), which the FDA green-lit in June. That approval came a year after the expansion of Alexion Pharmaceuticals (NASDAQ: AKXN) drug eculizumab (Soliris) to include NMOSD.

The Viela and Alexion drugs are delivered intravenously; the latter comes with a requirement for a program that communicate to physicians and patients the drug’s risks, which include the potential of associated meningococcal infections.

Genentech’s drug can be administered by an NMOSD patient or by a caregiver after training by a healthcare provider. The company said satralizumab, approved last Friday, is slated to become available in the US in two weeks. The drug, which is also approved in Canada, Japan, and Switzerland, is under review by regulators in the European Union and in China.