KalVista's Ekterly Wins FDA Approval as First Oral Treatment for Hereditary Angioedema Attacks
The U.S. Food and Drug Administration (FDA) has approved KalVista Pharmaceuticals' sebetralstat, to be marketed as Ekterly, for the treatment of acute hereditary angioedema (HAE) attacks in patients 12 years and older. This landmark decision marks the introduction of the first oral, on-demand treatment option for HAE, a rare genetic disorder characterized by painful and potentially life-threatening swelling attacks.
Breakthrough in HAE Treatment
Ekterly's approval represents a significant advancement in HAE management, offering patients a more convenient alternative to existing injectable therapies. Dr. Marc Riedl, an investigator in the pivotal trial from the University of California, San Diego, emphasized the importance of this development, noting that the oral administration could reduce delays in treating time-sensitive HAE attacks.
The drug's efficacy was demonstrated in the Phase III KONFIDENT study, which enrolled 136 patients. Results showed that Ekterly achieved symptom relief significantly faster than placebo, with median times of 1.61 hours and 1.79 hours for the 300-mg and 600-mg doses, respectively, compared to 6.72 hours for the placebo group.
Regulatory Journey and Market Implications
Ekterly's path to approval was not without challenges. The FDA initially delayed its decision beyond the June 17 target date, citing "heavy workload and limited resources." This delay sparked industry concerns about the impact of recent staff reductions at health agencies on regulatory timelines.
Despite these hurdles, KalVista has announced immediate availability of Ekterly in the U.S. market. The company has set a price of $16,720 per dose, consisting of two 600-milligram tablets. This pricing strategy, according to a KalVista spokesperson, reflects the drug's innovative nature and competitive positioning against existing therapies.
Competitive Landscape and Future Outlook
Ekterly enters a market currently dominated by injectable treatments such as Takeda's Firazyr and Pharming's Ruconest. Analysts estimate that Ekterly, KalVista's first marketed drug, could generate up to $600 million in annual U.S. sales at its peak.
The approval has been well-received by investors, with KalVista's shares rising 24% in early trading following the announcement. Stifel analyst Paul Matteis expressed optimism about Ekterly's potential, suggesting it could capture a significant share of the on-demand HAE market and potentially drive market growth.
As KalVista prepares for the commercial launch of Ekterly, the HAE treatment landscape continues to evolve. The recent approval of CSL's Andembry, a preventive HAE medication, underscores the ongoing innovation in this therapeutic area. With its unique oral formulation and promising efficacy data, Ekterly is poised to become a key player in the management of this rare but serious condition.
References
- KalVista Wins OK for First On-Demand Hereditary Angioedema Pill
Ekterly’s road to approval was not a smooth one. Last month, the FDA informed KalVista it would not meet its PDUFA date due to resource constraints and reports surfaced that Commissioner Marty Makary tried to have the application rejected.
- After delay, Kalvista wins FDA OK for drug to treat rare swelling disorder
The company had blamed FDA "resource constraints" for the delay, while a rival drug for hereditary angioedema received an on-time approval.
- KalVista bounces back from FDA delay with approval for oral rare disease med Ekterly
The FDA has approved sebetralstat under the brand name Ekterly as a new, oral treatment option for acute attacks of hereditary angioedema (HAE) in adults and children ages 12 years and older. The nod came just a few short weeks after the agency missed its June 17 target date originally established for the decision.
Explore Further
What are the detailed safety profiles and side effects observed during the Phase III KONFIDENT study for Ekterly?
What specific competitive strategies are companies like Takeda and Pharming employing in response to Ekterly's entry into the market?
How does Ekterly's pricing of $16,720 per dose compare with the cost of existing injectable HAE treatments?
What are the projected growth rates for the HAE treatment market following the introduction of Ekterly?
What innovations set Ekterly apart from recently approved preventive treatments like CSL's Andembry?