FDA Issues Draft Guidance on NTM-PD From MAC Studies

The FDA has laid out its draft recommendations for designs, study populations and endpoints for clinical trials of drugs designed to treat naïve and refractory nontuberculous mycobacterial pulmonary disease (NTM-PD) caused by Mycobacterium avium complex (MAC).

The concise agency draft guidance, which spans just six pages, offers sponsors considerations on the design and conduct of phase 1, 2 and 3 trials. In phase 1 and 2, the agency simply advises that it may be appropriate to delay therapy in certain patients, as long as there is proper monitoring, to support a short-term, randomized, placebo-controlled proof-of-concept study involving a single candidate.

For phase 3, the agency offers considerably more advice. For example, it advises that in general, sponsors should conduct two randomized, double-blind phase 3 trials, but it also notes that a single trial providing robust evidence of efficacy with confirmatory evidence may be enough to show substantial evidence of effectiveness.

Sponsors that do plan to seek approval based on a single trial and confirmatory data should discuss their development program with the FDA.