Alexion Pharmaceuticals’ drug Soliris has been approved to treat patients with a rare autoimmune disease that affects the central nervous system.
The U.S. Food & Drug Administration approved the drug for adult patients suffering from neuromyelitis optica spectrum disorder, or NMOSD, who are anti-aquaporin-4 (AQP4) antibody-positive.
Previously known as Devic’s Disease, it can cause progressive and irreversible damage to the brain, optic nerve and spinal cord. Patients with the disease are at risk for long term-disability, paralysis and blindness. The average age of first onset is 39 years, and the disease, which has symptoms similar to multiple sclerosis, primarily affects women. With this approval, Soliris is the first and only FDA approved treatment for the condition, according to Alexion.
Michael Levy, MD, a consultant to the company and associate professor of neurology at Massachusetts General Hospital in Boston, called it a “serious disease with devastating consequences.”
“Each attack can result in potentially irreversible consequences — causing blindness or losing the ability to walk — so preventing relapse is the primary goal of treatment,” Levy said.
According to Levy, with this approval, there is finally an FDA-approved treatment available for these patients.
The FDA approved the drug’s use for the disorder following a six-month review. According to an announcement from Alexion, at 48 weeks of treatment with Soliris, 98 percent of patients were relapse-free, compared to 63 percent taking a placebo.
John Orloff, MD, executive vice president and head of research & development at Alexion, said with NMOSD, disease progression is “driven by attacks,” which is why preventing any relapses is so important.
“For the first time, [these adult patients] have an approved treatment to reduce the risk of an unpredictable relapse — or attack,” Orloff said.
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Biohaven Pharmaceutical Holding Company Ltd. of New Haven has enrolled its first patient in a Phase 2 clinical trial of the safety and effectiveness of its drug rimegepant in treating trigeminal neuralgia.
The neuropathic pain disorder is characterized by frequent recurrent episodes of severe facial pain, which can result in significant disability.
The trial will take place at Johns Hopkins Medical Center.
Melissa Wolfe Beiner, MD, director of research and development and medical lead for the trigeminal neuralgia development program at Biohaven, said the company has worked closely with physicians at Johns Hopkins to advance this Phase 2 clinical trial.
“While trigeminal neuralgia is a rare disease, the patient burden associated with this disorder is significant,” Wolfe Beiner said in an announcement.
The drug rimegepant is the company’s lead candidate for the treatment of migraine.
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New Haven-based biotechnology company Arvinas Inc. has received FDA authorization to proceed with its investigational new breast cancer drug ARV-471.
The Phase 1 trial is expected to begin in the third quarter of 2019, and will assess the drug’s safety, side effects and anti-tumor effectiveness, according to a company announcement.
The drug is an oral estrogen receptor PROTAC protein degrader aimed at treating patients with locally advanced or metastatic breast cancer.
John Houston, president and CEO of Arvinas, said the company is pleased to be advancing the drug into the clinic.
“We hope the activity ARV-471 demonstrated preclinically will translate into a new, beneficial treatment for [these breast cancer] patients,” Houston said in an announcement.
According to the company, the drug in preclinical studies demonstrated positive results, including “robust tumor shrinkage.”
Contact Michelle Tuccitto Sullo at msullo@newhavenbiz.com
