DA approves Shire's Takhzyro for hereditary angioedema as ICER weighs in on cost effectiveness
(Ref: Channel News Asia, London South East, FDA, GlobeNewswire)
August 24th, 2018
By: Matthew Dennis
Tags: Top Story Cinryze Haegarda Ruconest Takhzyro CSL Pharming Shire Takeda FDA Marketing & Sales Regulatory Affairs
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Shire announced that the FDA approved Takhzyro (lanadelumab-flyo) for prophylaxis to prevent attacks of types I and II hereditary angioedema (HAE) in patients 12 years of age and older. Andreas Busch, head of R&D at Shire, noted that following clearance of the monoclonal antibody, which provides targeted inhibition of plasma kallikrein, people with HAE "have an innovative treatment that works differently than current options to help prevent attacks."
The approval was based on data from four clinical trials, including the Phase III HELP study, which showed that Takhzyro reduced the number of monthly HAE attacks an average of 87 percent versus placebo when administered at 300 mg every two weeks and 73 percent compared to placebo when administered at 300 mg every four weeks. Results of a pre-specified, exploratory analysis also showed that 44 percent of patients receiving Takhzyro 300 mg every two weeks had zero attacks compared to placebo for the 26-week treatment period.
Shire noted that Takhzyro has a half-life of approximately two weeks and is administered as one subcutaneous self-injection every two weeks at the recommended starting dose. The recommended starting dose of the therapy is 300 mg every two weeks, while a dosing interval of 300 mg every four weeks may be considered if the patient is well-controlled for more than six months.
Shire gained Takhzyro via its acquisition of Dyax in 2016 for around $5.9 billion. The company noted that following approval of Takhzyro, Dyax shareholders will receive $4 per share in cash as part of the contingent value right received through the transaction. The drug is also under review in Europe and Canada, where regulators accepted filings for the therapy in March.
Takhzyro is also one of the key reasons behind Takeda agreeing to acquire Shire earlier this year for around $62 billion. Analysts at Equity Research BTIG forecast peak sales of about $1.4 billion to $1.5 billion for the drug within five years of launch, while Jefferies analysts said it "could well become the dominant therapy in [hereditary angioedema]," with revenue of approximately $1.8 billion.
The approval of Takhzyro came the same day as the Institute for Clinical and Economic Review (ICER) released a draft report into the comparative clinical effectiveness and value of therapies for the long-term prophylaxis against hereditary angioedema attacks. The ICER looked at Takhzyro, along with the three C1 inhibitors comprising CSL Behring's Haegarda, Pharming's Ruconest and Shire's Cinryze.
The ICER noted that the net price per package for Haegarda is $1393, Cinryze costs $2725 and Ruconest is priced at $4230, while pricing information was not available at the time for Takhzyro, with the agency using an estimated cost of $19 447. According to the assessment, the price of the drugs to achieve $100 000 per quality-adjusted life year (QALY) is $1131 for Ruconest, $1193 for Cinryze, $1540 for Haegarda and $15 601 for Takhzyro. ICER concluded that Haegarda was "dominant" over no prophylaxis, while Cinryze and Ruconest were "unlikely" to be cost-effective at the same cost per QALY gained.
For related analysis, see ViewPoints: As lanadelumab PDUFA nears, CSL provides bullish update for Haegarda.
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