Lilly’s CGRP Emgality and Spark’s Luxturna win CHMP backing, as Sarepta’s Exondys misses

EUDRA
The CHMP endorsed 13 medicines for approval, nixed Sarepta’s controversial DMD therapy Exondys, and backed seven drugs’ indication expansion applications. (EMA)

The European Medicines Agency’s (EMA) drug review panel is on fire this week. It recommended 13 medicines—including Eli Lilly’s CGRP player Emgality—for approval, nixed Sarepta’s controversial Duchenne muscular dystrophy therapy Exondys after re-examination and backed seven drugs’ indication expansion applications.

With a positive opinion from the Committee for Medicinal products for Human Use (CHMP) for EU approval, Eli Lilly’s CGRP migraine injection Emgality, also known as galcanezumab, is now one step closer to challenge Amgen and Novartis’ first-in-class Aimovig, which just got its official EU nod two months ago.

In three pivotal studies, Emgality on average helped patients with episodic or chronic migraine cut the monthly time they had experienced migraine by 1.9 days and 2 days, respectively.

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Fierce competition in the CGRP field is quickly building up. As Emgality still awaits an FDA decision, which could come soon in the coming days, Aimovig and Teva’s just-approved Ajovy have already landed in the U.S., both at the price of $6,900 a year. There’s at least a lot of demand for Aimovig, and the initial interest is “beyond our expectations,” said Amgen commercial chief Anthony Hooper on the company’s second-quarter earnings call.

RELATED: Teva, chasing Amgen's Aimovig, scores much-needed FDA approval for migraine drug Ajovy

Last December, Spark Therapeutics’ Luxturna made history as the first FDA-approved gene therapy that directly administers a healthy gene to replace a malfunctioned one. Now, European patients suffering from inherited retinal dystrophy caused by RPE65 gene mutations might soon have access to it as well.

Through a licensing deal signed in January, Luxturna will be sold in Europe by Novartis, who paid Spark $105 million upfront and offered $65 million in milestones. In the U.S., Spark is charging a staggering $850,000 for the drug, a price that came within analysts’ expectations but still drew criticism from patient advocates. But the company promised not to collect the full cost if the drug doesn’t work.

Bayer’s long-acting hemophilia treatment Jivi and Kyowa Kirin’s Poteligeo for two rare types of non-Hodgkin lymphoma were the other two orphan medicines that got endorsement from the agency, hard on the heels of their FDA nods in August.

In addition, Melinta’s antibiotic Vabomere, Merck & Co.’s HIV therapies Pifeltro (doravirine) and doravirine-based triplet Delstrigo, Takeda’s ALK-positive lung cancer drug Alunbrig, and three biosimilar versions of Amgen’s Neulasta from Mylan, Cinfa and Sandoz were also winners at the panel this week.

RELATED: Novartis’ and Gilead’s CAR-Ts, Alnylam’s Onpattro and others rack up EU approvals

However, the cheerful mood wasn’t captured by Sarepta. Controversy accompanied the company’s Duchenne muscular dystrophy therapy Exondys 51 through its regulatory process before the FDA, which approved the drug against the recommendation of an expert panel. Opinions were widely split even within the FDA, as its former new drugs director John Jenkins had publicly criticized the agency’s decision.

It seems like the FDA’s European counterpart wasn’t so persuaded by the drug’s sparse clinical evidence. After giving a thumbs-down to a conditional marketing application in May, the CHMP has again shot it down. Sarepta CEO Doug Ingram said in a statement that the negative opinion, though largely anticipated, was disappointing for the team. 

But as Leerink analyst Joseph Schwartz sees it in a Friday memo to investors, the drug’s approval in the EU is just a matter of “when” not “if.” The company now looks for scientific advice next year to explore a potential path forward for Exondys in Europe, and Schwartz noted that a phase 3 trial dubbed PROMOVI has completed enrollment with a primary completion date of May 2019 that could resolve regulators’ questions or concerns.

Meanwhile, several drugs gained indication expansion recommendations from CHMP.

Despite debate over its trial design, GlaxoSmithKline’s Trelegy followed an approval in the U.S. and nabbed a positive opinion that will soon allow it tap into COPD in Europe. Novartis’ Gilenya can now expect to treat children with multiple sclerosis. The agency also suggests AbbVie’s Venclyxto, when paired with rituximab, can serve as a second-line therapy for patients with relapsed or refractory chronic lymphocytic leukemia (CLL). And Astells’ Xtandi won positive opinion to treat men with high-risk non-metastatic castration-resistant prostate cancer.

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