New Treatment For Alpha-1 Antitrypsin Deficiency Lung Disease Has Been Granted Orphan Drug Designation

Mereo BioPharma Group has just announced that they have received Orphan Drug Designation from the FDA for their new treatment for alpha-1 antitrypsin deficiency (A1AD) called alvelestat. This therapy is specifically for A1AD-lung disease or A1AD-LD.

The company is grateful to have received this designation, which provides marketing exclusivity for 7 years following approval, expedited development, and various financial incentives.

They hope to provide an additional update on alvelestat in the next two months.


A1AD is a rare disease caused by a deficiency of AAT, an essential protein within our livers. This protein is responsible for protecting the lungs from inflammatory enzymes (including neutrophil elastase). Ultimately, patients face lung damage from elastin destruction. They also may face liver disease from abnormal AAT located in the liver.

A1AD patients can face pulmonary emphysema which is a chronic and progressive lung disease. Symptoms can include asthma, a chronic cough, wheezing, chest infections, trouble breathing, bronchiectasis, and sputum production.

Since the lung disease is progressive, symptoms often start early in adulthood and as time goes by, patients frequently necessitate oxygen on a daily basis. For the most extreme cases, a lung transplant is necessary.


Alevestat or, MPH-966, is an oral medication which inhibits neutrophil elastase (NE).

Currently in progress is a Phase 2 clinical trial for alevestat in those with severe A1AD. Researchers are hopeful that this treatment could prevent additional lung damage for patients.

Additionally, there is a study currently ongoing for AATD-LD. This study is funded by NCATS.

Finally, there are Phase 1b/2 studies of alvelestat for COVID-19 as well as bronchiolitis obliterans syndrome (BOS).

You can read more about this new orphan drug designation and the development of alvelestat here.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email