FDA approves Rystiggo for adults with generalized myasthenia gravis
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The FDA has approved Rystiggo, a subcutaneous infusion therapy to treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive.
According to a press release from the developer of the drug, UCB, Rystiggo (rozanolixizumab-noli) is a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor and reduces circulating IgG. It is the only FDA-approved treatment for adults with both anti-AChR and anti-MuSK antibody-positive generalized myasthenia gravis, the two most common subtypes of the disease, the company said.
“No two people living with [generalized myasthenia gravis] experience the disease in the same way, so we can’t take a ‘one-size-fits-all’ approach to disease management,” Iris Loew-Friedrich, UCB executive vice president and chief medical officer, said in the release. “Disease management should be based on the clinical needs and preferences of the individual patient, and the aim of treatment is to help restore that patient’s ability to carry out activities of daily living.”
FDA approval was granted under Priority Review and was based on data from the multicenter, randomized, double-blind, placebo-controlled phase 3 MycarinG study, in which 200 adults received rozanolixizumab-noli (7 mg/kg or 10 mg/kg) or placebo for 6 weeks.
The study met its primary endpoint of comparative change from baseline between treatment groups in MG Activities of Daily Living Scale (MG-ADL) total score at day 43, with either dose of rozanolixizumab-noli outperforming placebo (–3.4 points vs. –0.8), the release stated. In addition, the secondary endpoint of comparative change from baseline to day 43 in Quantitative Myasthenia Gravis score (QMG) also favored rozanolixizumab-noli (–5.4 points in the 7 mg/kg group and –6.7 points in the 10 mg/kg group vs. –1.9 points in the placebo group).
The injectable was generally well-tolerated, the company said, with the most common adverse reactions including headache, infections, diarrhea, pyrexia, hypersensitivity reactions and nausea.
“[Generalized myasthenia gravis] can cause unpredictable fluctuations in severity and frequency of symptoms, which are often debilitating and can substantially impact the lives of patients,” Vera Bril, BSc, MD, FRCPC, professor of neurology at the University of Toronto and lead investigator of the MycarinG study, said in the release. “There is a significant need for new, innovative treatment options to reduce the day-to-day burden of [generalized myasthenia gravis].”
According to the release, rozanolixizumab-noli will be commercially available in the U.S. in the third quarter of 2023. It received orphan drug designation from the FDA in 2019.