- A targeted cancer therapy from Bayer and a seizure medication from GW Pharmaceuticals have won over the committee responsible for recommending what drugs should be cleared for the European market, though with a couple important caveats.
- The committee, often referred to by its acronym CHMP, recommended conditional approval of Bayer's Vitrakvi, meaning the company will at some point need to provide additional data supporting the drug's efficacy. Vitrakvi targets a genetic alteration often found in certain rare solid tumors, and is the first cancer drug to receive a CHMP recommendation not defined by tumor location.
- As for GW's Epidiolex, the CHMP supported its use in seizures associated with two uncommon syndromes — but as adjunctive therapy to clobazam, a standard of care drug for orphan epilepsies. That's a departure from U.S. regulators, who last year approved Epidiolex by itself for the indication. Stifel analyst Paul Matteis called the CHMP's opinion "disappointing," but concluded the clobazam provision wouldn't significantly affect Epidiolex sales.
Bayer secured full rights to Vitrakvi (larotrectinib) from Loxo Oncology in February, about a month after the biotech announced it was being acquired by Eli Lilly.
The drug only recently entered the U.S. market, and has yet to creep onto Bayer's list of best-selling drugs. Still, Vitrakvi represents a valuable milestone in cancer research as the second-ever cancer drug approved for patients with a select DNA alteration rather than certain tumor types.
Epidiolex (cannabidiol) also brought a noteworthy approval, as the first cannabis-based drug to clear the Food and Drug Administration.
Indicated for patients with two uncommon forms of epilepsy called Lennox-Gastaut syndrome and Dravet syndrome, net sales of Epidiolex have already surpassed Wall Street expectations for the short period it's been on the market. They totaled $33.5 million in the first quarter, which was more than double what analysts had predicted.
Now, Epidiolex looks primed to break into the European market as well. The European Union typically follows the CHMP's recommendations.
Stifel predicts the European market will account for 15% of Epidiolex's sales by 2023, while ex-U.S. sales will account for roughly 17% by 2025. That 17% would reflect about $400 million of the roughly $2.4 billion the investment bank forecasts for Epidiolex's 2025 sales.
While noting that the CHMP's opinion regarding Epidiolex is more "conservative" than the monotherapy approval granted in the U.S., Stifel's Matteis doesn't think it will have too much bearing on the drug's performance.
"What exactly this means for uptake is unclear because we don't know of any good data source that codifies exactly how much clobazam is used in Europe," he wrote in a July 26 note to clients.
"[T]hat being said, as we understand it, clobazam has been generic in Europe for a long time and is a staple of the Dravet/LGS/orphan epilepsy standard-of-care," he added. "It's plausible too that some physicians could — if they want to prescribe Epidiolex to a currently non-clobazam treated patient — simply write the drug alongside a low clobazam dose."
The CHMP, short for the Committee for Medicinal Products for Human Use, recommended five drugs for approval during its July meeting.
In addition to Vitrakvi and Epidiolex, the committee backed Acorda Therapeutics' Inbrija (levodopa) for symptoms of 'off' periods in Parkinson’s disease, Theratechnologies' Trogarzo (ibalizumab) for HIV, and Mylan's generic deferasirox for chronic iron overload in patients with certain anemias.