Alexion Pharmaceuticals said this week it will ask federal regulators to greenlight its freshly approved blood-disorder drug Ultomiris for the treatment of a second ultra-rare disease.
The New Haven-born biotech, which still has a significant research presence in the city, plans to submit an application to the U.S. Food & Drug Administration by June to market the drug for the treatment of the blood disease Atypical Hemolytic Uremic Syndrome (aHUS).
The announcement follows promising results in a phase 3 clinical trial of patients with the disease, which is characterized by inflammation and blood clotting in small blood vessels throughout the body. Causing progressive organ damage and kidney failure, the condition is often fatal.
Alexion said nearly 54 percent of patients in the study responded fully after 26 weeks of treatment. Patients were considered fully responsive if they experienced normalized platelet counts, lower red blood cell destruction and improved kidney function at least once for 28 consecutive days during treatment.
“We are very pleased with these data, which demonstrate that Ultomiris can provide clinically meaningful benefits to patients with aHUS,” Alexion R&D head John Orloff, MD said in a statement. “We are preparing regulatory submissions for Ultomiris in aHUS in the U.S., European Union and Japan as quickly as possible.”
In December the FDA approved Ultomiris to treat another ultra-rare blood disease, paroxysmal noctural hemoglobinuria (PNH).
With a more convenient dosing schedule calling for intravenous infusions every eight weeks instead of every two, Alexion has said Ultomiris could replace its blockbuster Soliris as the standard of care for PNH.
Alexion moved its headquarters to Boston last summer.
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The FDA has revived clinical trials for Biohaven Pharmaceuticals Holding Company Ltd.’s new drug to treat the neurodegenerative disease multiple system atrophy (MSA).
Biohaven asked the FDA to reactivate an investigational new drug application (IND) initially filed by pharma giant AstraZeneca, which recently licensed the compound to the New Haven startup.
The drug, known as BHV-3241, is a first-in-class, oral MPO (myeloperoxidase) inhibitor. MPO is an enzyme linked to brain inflammation, which plays a role in MSA and other neurodegenerative diseases.
“We believe BHV-3241 has the potential to be the first effective treatment for people living with MSA and expect to start our phase 3 trial in the third quarter of 2019,” said Biohaven CEO Vlad Coric, MD.
MSA is a rare, progressive disorder that causes Parkinson’s disease-like movement problems such as tremors and poor balance, as well as problems with involuntary functions like blood pressure, bladder control and digestion. Patients usually die within six to 10 years of onset.
Contact Natalie Missakian at news@newhavenbiz.com
