New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress

Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia (PA) and methylmalonic acidemia (MMA).

MMA and PA are caused by enzyme deficiencies which inhibit the body’s ability to metabolize their amino acids normally. This leads to a buildup of metabolites in the body which cause organ damage, developmental deficits, seizures, and ultimately, early death.

There are currently no truly effective treatment options for either of these conditions and patients are desperate for new options.


But what do these designations mean? Fast Track Designation helps incentivize the rapid development of a novel therapy which could provide extreme benefit to patients who currently have no or limited options. It increases communication with the drug development company and the FDA, allows for priority review of their application, and provides accelerated approval for the company’s eventual New Drug Application.

Rare Pediatric Disease Designation is specifically allotted to investigative therapeutics targeting rare diseases that primarily affect children. It also provides priority review for the company’s application.

HST5040 Therapy

HST5040 is an oral therapy that is taken once each day. Researchers hope it will help to treat the metabolic abnormalities that arise from MMA and PA.

This therapy is a small molecule meaning it can be easily distributed across multiple tissues including the brain, the muscles, and the heart.

It can be taken through the mouth or by using a feeding tube.

Phase 2 Trial

HemoShear was authorized to begin a Phase 2 trial of this therapy in June.

The Phase 2 clinical trial will include 12 patients who are diagnosed with either PA or MMA. It is called HERO which stands for HElp Reduce Organic Acids.

You can read more about these new designations and clinical trial here.

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