Amicus Therapeutics scores another approval in the UK for Pompe disease dual therapy

The Medicines and Healthcare products Regulatory Agency (MHRA) in the UK has granted a marketing authorization for Amicus Therapeutics’ combo therapy for late-onset Pompe disease, a rare inherited lysosomal disorder, the company announced Tuesday.

The drugs, marketed as Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat), have also been recommended as first-line as well as later lines of therapy for the disease by the National Institute for Health and Care Excellence (NICE). Pombiliti is a long-term enzyme replacement therapy while Opfolda is an enzyme stabilizer.

The drugs are also known as AT-GAA.

Bradley Campbell

“The speed in which NICE recommended reimbursement of Pombiliti and Opfolda is reflective of the U.K.’s Innovative Licensing and Access Pathway, the data behind Pombiliti and Opfolda, the strong collaboration with the reimbursement authorities, and the Amicus commitment to bring this therapy to those living with Pompe disease as quickly as possible,” Amicus president and CEO Bradley Campbell said in a statement.

The drug combo has already been approved in the EU, and Amicus has started its commercial launch in Germany while the company has “reimbursement discussions” with other EU countries, according to Amicus’ second quarter earnings report.

Amicus added that it also expects a US approval in the third quarter of 2023 after the company announced in March that the FDA had scheduled a pre-approval inspection. The FDA has delayed its approval of the therapy several times, which includes a BLA for cipaglucosidase alfa and an NDA for miglustat.

In May 2022, the company said that the FDA had pushed back its review period by 90 days. At the time, Amicus said the FDA was still reviewing newly submitted information as part of ongoing reviews, but that it is not related to requests for additional clinical data.

Then in October that year, Amicus revealed that the FDA had again delayed an approval decision needed for AT-GAA after the agency was unable to complete a manufacturing inspection in China because of travel disruptions. That inspection has since been completed.

Leerink analysts wrote in an August note that company management said it was “pleased” with the inspection of the China manufacturing facility, and that it didn’t believe any additional inspections will be needed. Leerink is predicting US sales of the therapy of $8.8 million, down from $9.7 million, assuming there are about 64 patients on commercial therapy at the end of the year. Leerink is also predicting 2023 EU sales of $10 million if approximately 72 patients are on the therapy by the end of the year.

There was also some doubt around the Phase III data submitted with the company’s applications: The combination did not beat Lumizyme (alglucosidase alfa), Sanofi’s enzyme replacement therapy, in its Phase III PROPEL trial. It did not hit statistical significance on the primary endpoint (mean change in 6-minute walk distance), earning a p=0.072.

In the 52-week study, patients saw a “nominally” statistically significant and clinically meaningful improvement in respiratory function. There were also improvements in muscle strength, coordination, mobility and fatigue (most were not statistically significant), so Amicus pushed forward with the filing anyway.

Amicus did tout longterm results of the drug this year from its extension PROPEL study, including “durable mean improvements” in a six-minute walk distance, stable lung function and reductions in muscle damage in patients who were on the therapy for up to 104 weeks.

manufacturing
therapeutics
healthcare
authorization
fda