Amicus nabs EU nod for Pompe disease newcomer Pombiliti, setting up fight with Sanofi

After a COVID-19-related inspection delay sidelined Amicus Therapeutics' approval hopes in the U.S., the company's CEO said it was a matter of “when," not "if," its rare disease candidate AT-GAA would score a green light in Pompe disease.

Monday, the company largely proved its thesis in Europe.

Amicus’ drug—which combines a long-term enzyme replacement therapy (ERT) with the oral enzyme stabilizer miglustat—won an approval from the European Commission as Pombiliti in adults with late-onset Pompe disease (LOPD). Now, the company must wait on the European Medicines Agency’s Committee for Medicine Products for Human Use to deliver a verdict on miglustat specifically, which it expects to come in the second quarter of 2023, Amicus said in a release.

Once both components of the drug are approved, the company will work to bring Pombiliti to LOPD patients in Europe “as rapidly as possibly," CEO Bradley Campbell said in a statement.

Pompe disease is a rare neuromuscular disorder that leads to progressive weakness in the heart and skeletal muscles. It is caused by mutations in a gene that makes the enzyme acid alpha-glucosidase, which the body uses to break down the stored sugar glycogen.

With a broad label that covers “all adults” in Europe with LOPD, plus the company’s self-professed “launch readiness,” Campbell has flagged a “significant” commercial opportunity for his drug overseas. Pombiliti could become the next standard of care in the field, the CEO said in a statement.

Once Pombiliti hits the market—potentially sometime in the second half of the year—analysts at Berenberg Capital Markets expect the two-part therapy to siphon market share from Sanofi’s decade-old Pompe drug Lumizyme. Amicus’ drug’s prospects against Sanofi’s new iteration on its med Nexviazyme remain more “unclear,” the analysts wrote in a note to clients this past winter.

Nexviazyme, for its part, netted 196 million euros in global revenues last year. Sanofi is also positioning its successor med as a new standard of care in Pompe disease.

As for Pombiliti, Berenberg analysts expect the drug to generate some $33 million in 2023 sales.

The European Commission granted approval to Pombiliti based on results from the phase 3 study PROPEL, which Amicus says is the only LOPD trial to include patients in the ERT-experienced patient population, where there’s a high unmet need for new treatments. Amicus also tested its drug in LOPD patients who hadn’t previously tried ERT.

The European win comes after a slight regulatory hiccup for AT-GAA in the U.S.

Back in October, the FDA delayed Amicus’ biologics license application for cipaglucosidase alfa after COVID-19 travel restrictions thwarted a planned inspection of a Chinese manufacturing site operated by CDMO WuXi Biologics. At the time, the FDA didn’t provide a new target action date for the potential approval, which had originally been set for Oct. 29, 2022.

In light of the delay, Amicus said it was “actively engaged” with the regulator to develop plans and hammer out logistics for a preapproval inspection scheme. Amicus stressed that the pandemic travel snafu was the “sole reason” for the FDA’s action date extension.

Since then, the U.S. Food and Drug Administration has rescheduled the preapproval inspection, with the U.S. approval decision expected around 2023’s third quarter, pending a successful site tour, an Amicus spokesperson said over email.