Dive Brief:
- The Food and Drug Administration has set back Pharming Group's hopes to expand the approval of its hereditary angioedema (HAE) drug Ruconest, issuing the Dutch company a complete response letter just ahead of the target action date of September 21.
- Pharming's supplemental Biologics License Application to the FDA was supported by Phase 2 data. The regulator wasn't moved, however, and asked Pharming to conduct an additional clinical trial to further evaluate Ruconest's efficacy as a preventive treatment for HAE.
- Bruno Giannetti, chief operating officer at Pharming, said the company will work with the FDA to "generate additional clinical data required to enable access for patients to use Ruconest for HAE prophylaxis."
Dive Insight:
Hoping to be the first to hit the market from both prevention and treatment of HAE, Pharming submitted a supplemental Biologics License Application for its recombinant human C1 esterase inhibitor Ruconest (conestat alfa) in November 2017.
That won't be in the cards in the near term, however, as Pharming will now need to conduct further study.
"We see this as a minor setback. Pharming remains committed to serving the HAE community. We will continue and have the resources to develop new innovative and more convenient administration options of Ruconest for acute treatment and prophylaxis of HAE," said Sijmen de Vries, CEO of Pharming, in a statement.
Ruconest was approved for acute treatment of HAE in July 2014, and Pharming reacquired the U.S. rights from Valeant Pharmaceuticals in August 2016.
In the meantime, the HAE space looks to becoming more competitive. Shire recently won approval for prophylactic use of its drug Takhzyro (lanadelumab), which takes a different approach to Ruconest by targeting plasma kallikrein.
Last month, the Institute for Clinical and Economic Review (ICER) released a draft evidence report on long-term treatments for prevention of hereditary angioedema attacks that said that the "annual potential budgetary impact of treating the entire eligible population over five years did not exceed the $991 million ICER budget impact threshold at any price level, with Ruconest approaching the threshold (97%) at current WAC [wholesale acquisition cost of $830,616], largely due to the relatively small number of patients eligible for treatment."
Takhzyro also came in under the threshold, although the report only used a placeholder list price of $537,097, considerably lower than Ruconest.