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US FDA grants Breakthrough Therapy de...
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US FDA grants Breakthrough Therapy designation to Dyax’s DX-2930 for prevention of HAE attacks
Pharma News

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Dyax Corp’s DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKal), being developed as a subcutaneous injection for prevention of hereditary angioedema (HAE) attacks.

Uncontrolled pKal activity leads to excessive generation of bradykinin, a vasodilator thought to be responsible for the localised swelling, inflammation and pain characteristically associated with HAE.

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 organisational 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 programme,” 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 maximise the possibility of a rapid path to approval.”

HAE is a rare acute inflammatory condition characterized by episodes of severe, often painful swelling affecting the extremities, gastrointestinal tract, genitalia, and larynx. HAE is caused by low or dysfunctional levels of C1 esterase inhibitor (C1-INH), a naturally occurring molecule that inhibits plasma kallikrein, a key mediator of inflammation, and other serine proteases in the blood. HAE is estimated to affect up to 1 in 50,000 individuals.

Dyax is a biopharmaceutical company focused on the development and commercialisation of novel biotherapeutics for unmet medical needs.

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