RedHill Biopharma announces QIDP Fast-Track Designation granted by FDA to RHB-104 for Nontuberculous

Under FDA's GAIN Act, QIDP designation allows for Fast-Track status and Priority Review, potentially leading to a shorter NDA review time by the FDA, and, if approved, an additional five years of U.S. market exclusivity on top of the standard exclusivity period.

RedHill Biopharma Ltd., a specialty biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, has announced that RHB-104 has been granted Qualified Infectious Disease Product (QIDP) designation by the U.S. FDA for the treatment of Nontuberculous Mycobacteria (NTM) infections. The QIDP designation was granted under the FDA’s Generating Antibiotic Incentives Now (GAIN) Act, which is intended to encourage development of new antibiotic drugs for the treatment of serious or life-threatening infections.

The granted QIDP designation allows RedHill to benefit from the Fast-Track status, with an expedited development pathway for RHB-104 for the treatment of NTM infections, as well as Priority Review, which provides for a shorter review time by the FDA of a future potential marketing application. If approved for the treatment of NTM infections, RHB-104 would also receive an additional five years of U.S. market exclusivity on top of the standard exclusivity period.

RHB-104 is a proprietary and potentially groundbreaking antibiotic combination therapy in oral capsule formulation, with potent intracellular, anti-mycobacterial and anti-inflammatory properties.

In light of the QIDP designation, RedHill will consult with the FDA regarding its RHB-104 development program for NTM infections.

NTM infections can occur throughout the body, although pulmonary infections, lymphadenitis, and skin and soft tissue infections are the most commonly affected areas. There are a number of risk factors that can increase a person’s chances of acquiring NTM infections, including history of bronchiectasis, chronic obstructive pulmonary disease, smoking, HIV/AIDS, use of immunosuppressive drugs and certain genetic conditions, such as cystic fibrosis and alpha-1 antitrypsin deficiency.

Nontuberculous mycobacteria are ubiquitous organisms commonly isolated from the environment and identified in drinking water and soil. Patients suffering from NTM infection are at increased risk for reinfection. NTM infections have been increasing worldwide over the past two decades, while treatment of NTM infection remains difficult and entails multiple antibiotics and an extended treatment course due to the risk of development of resistance.

A first Phase III study with RHB-104 for the treatment of Crohn’s disease (the MAP US study) is currently ongoing in the U.S. and additional countries. The development of RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease, and potentially other autoimmune diseases, are related to Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients.

Recently, RedHill also completed a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in patients treated for relapsing remitting multiple sclerosis (the CEASE MS study). Top-line final results from the CEASE MS study suggest meaningful positive safety and clinical signals upon 24 weeks of treatment with RHB-104 as an add-on therapy, thereby supporting further clinical development.