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July 16, 2021

Alexion drug meets goal in autoimmune disease study

PHOTO | Contributed Alexion, AstraZeneca Rare Disease's research facility at 100 College St. in New Haven.
By Natalie Missakian

New Haven-born Alexion Pharmaceuticals’ Ultomiris met the main goal of a late-stage human trial on adults with a rare autoimmune disease, putting the new drug closer to its third FDA approval.

Alexion on Thursday announced the positive results in a study of the drug on 175 patients with generalized myasthenia gravis (gMG), a disease marked by severe muscle weakness.

The disease is caused by inflammation that damages the connection point between nerve cells and the muscles they control, leading to a breakdown in communication between the brain and the muscles.

Initially patients may experience slurred speech, droopy eyelids, double vision and lack of balance, but the disease can progress to include more severe symptoms like choking, impaired swallowing, extreme fatigue and episodes of respiratory failure.

Alexion’s blockbuster drug Soliris was the first new treatment in 60 years for people with severe symptoms of the disease, the company said. 

But with Ultomiris, Alexion aims to help a broader swath of gMG patients, including those who have milder symptoms or are earlier in their treatment, R&D head John Orloff said in a statement. Soliris is only approved for severe cases.

“These data provide confidence that Ultomiris  has the potential to become the new standard of care for gMG and may reduce patient burden with its less frequent dosing schedule [compared to Soliris],”  Orloff said.

Alexion said gMG patients in the Phase 3 study who took Ultomiris saw a significant improvement in being able to carry out activities of daily living after 26 weeks, based on scores in a patient-reported assessment, compared to those taking a placebo.

The drug already has FDA approval for the rare blood disorder PNH (paroxysmal nocturnal hemoglobinuria), and aHUS (atypical hemolytic uremic syndrome), an ultra-rare disease that causes kidney damage. 

Based on the study, Alexion said it is planning regulatory filings for the new indication in the U.S., European Union and Japan by the end of this year or early 2022.

Thursday’s announcement comes as the biotech, which has more than 500 employees in New Haven, is set to become the rare disease arm of AstraZeneca next week.  

The pharma giant expects to close on its $39 billion acquisition of the company July 21.

Contact Natalie Missakian at news@newhavenbiz.com

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