The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigation of DX-2930 for HAE. Dyax Corp. is developing DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKal), as a subcutaneous injection for prevention of HAE attacks.
Breakthrough Therapy designation is intended to expedite the development and review of potential new medicines with early signal of clinical benefit in serious or life-threatening conditions and helps ensure patients have access to them as soon as possible. Breakthrough Therapy designation is considered when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy. The benefits of Breakthrough Therapy designation include organizational commitment involving the FDA’s senior managers and with more intensive guidance. Breakthrough Therapy designation does not change the standards for approval.
The designation is supported by the interim results of Dyax’s Phase 1b clinical trial of DX-2930 in HAE patients. The Phase 1b study met all objectives assessing safety, tolerability and pharmacokinetics of multiple subcutaneous administrations of DX-2930. Additionally, in a pre-specified proof-of-concept efficacy analysis, DX-2930 demonstrated statistically significant reductions in attack rate compared to placebo.
“Receipt of Breakthrough Therapy designation is a key milestone for the DX-2930 development program,” said Burt Adelman, M.D., Executive Vice President of Research and Development and Chief Medical Officer at Dyax. “We look forward to taking full advantage of the opportunities that Breakthrough Therapy designation allows in order to maximize the possibility of a rapid path to approval.”