Orphan Drug Designation Granted to Sickle Cell Medication

The FDA recently granted orphan drug designation to Imara, Inc’s IMR-687 to treat patients with sickle cell disease.

IMR-687 was designed to attack the pathology of sickle cell disease, and is a selective phosphodiesterase 9 (PDE9) inhibitor, according to a press release. The drug has the potential to treat sickle cell diseases and other hemoglobinopathies.

Preclinical trials have revealed that IMR-687 can reduce the sickling of red blood cells, and blood vessel occlusion that leads to pain, organ damage, and early death in patients with sickle cell disease.

Currently, the safety and pharmacokinetics of IMR-687 are being explored in a phase 1a clinical trial of healthy patients. Imara also plans to determine the pharmacodynamics of the drug.

Primary outcomes include the proportion of patients experiencing adverse events and changes in vital signs from baseline. Investigators will measure blood pressure, heart rate, pulse, and temperature.

Additionally, the researchers will determine other hematologic or physical changes from baseline.

There are limited treatment options for patients with sickle cell disease. They may be treated with blood transfusions, bone marrow transplants, or drugs, but there is no cure. As such, more effective treatment options are needed for patients with this rare disease.

“This designation is granted to investigational new drugs that have shown promise to address serious medical needs for patients living with rare conditions,” said James McArthur, PhD, founder, president, and chief executive officer of Imara. “This is an important milestone for Imara, and we look forward to continuing our efforts to advance potential new treatments for patients.”