Principia Biopharma Announces PRN1008 Receives Orphan-Drug Designation from FDA

Principia Biopharma (Nasdaq:PRNB), a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, today announced that PRN1008, an oral, reversible covalent Bruton’s Tyrosine Kinase (BTK) inhibitor, has been granted orphan-drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with immune thrombocytopenic purpura (ITP).

As quoted in the press release:

“The FDA granting orphan-drug designation is an important milestone as we continue to develop PRN1008 for ITP patients in need,” said Martin Babler, Chief Executive Officer of Principia. “We believe that PRN1008 has the potential to modulate the immune system’s attack on platelets, the underlying cause of ITP, and to be an effective oral therapy to reduce the untoward effects often seen with more invasive or broader immune suppression.”

The FDA’s orphan-drug designation program incentivizes the development of products intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 people in the U.S. Among the benefits of being the first sponsor of a designated orphan drug to receive FDA marketing approval for such a rare disease or condition include seven years of marketing exclusivity, waiver or partial payment of fees prescribed by the Prescription Drug User Fee Act, and tax credits for qualified clinical research costs relating to such designated orphan product.

Click here to read the full press release.