Pombiliti plus Opfolda approved for LOPD in the UK

Amicus' two-part therapy is for adults with late-onset Pompe disease

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat), a two-part therapy formerly known as AT-GAA, has been approved by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) as a treatment for adults with late-onset Pompe disease (LOPD).

“The MHRA approvals for Pombiliti and Opfolda are a major step forward for adults in the U.K. living with late-onset Pompe disease who are seeking new treatments,” Bradley Campbell, president and CEO of the two-component therapy’s developer Amicus Therapeutics, said in a press release.

Amicus also announced that the National Institute for Health and Care Excellence (NICE) has issued final guidance recommending that Pombiliti plus Opfolda should be reimbursed by the National Health Service (NHS) in England and Wales. NICE concluded that the combination therapy was cost-effective based on its expected benefit for patients.

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Pombiliti plus Opfolda previously had received U.K. designations that are intended to bring new therapies to market faster, including an innovation passport under the innovative licensing and access pathway.

“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,” Campbell said.

He added: “I am proud of Amicus’ relentless commitment toward ensuring patient access to our innovative therapies, and we are working as quickly as possible to make Pombiliti and Opfolda commercially available.”

“Pompe disease is a rare disease, one that extensively affects all aspects of life for an individual and their family, friends, and colleagues,” said Allan Muir, chair of the board of trustees at the Pompe Support Network. “We are very grateful to companies such as Amicus Therapeutics that seek to make meaningful differences for our small community and we welcome new treatments that bring further choice to patients.”

Pombiliti and Opfolda meet Great Britain’s orphan designation criteria, meaning both will benefit from a guaranteed decade of market exclusivity. Prior to the approval, the combination therapy had been available in the U.K. through an early access program.

‘Exciting development’ for the late-onset Pompe community

“From the positive uptake of the Early Access to Medicines Scheme, we have already seen the impact that this treatment is having for patients,” said Mark Roberts, MD, a neurologist at Salford Royal NHS Foundation. “Having widespread access to this treatment is an exciting development for the Pompe community, giving [healthcare providers] and patients a new option that exhibits a novel mode of action.”

Val Buxton, chief executive of the Association for Glycogen Storage Diseases, added: “Being able to gain rapid access to effective new therapies via the Early Access to Medicines Scheme has had a meaningful impact on many people affected by Pompe. This approval has provided a much-needed new therapy option for the Pompe community.”

Pompe disease is caused by mutations that interfere with the production of the enzyme acid alpha-glucosidase. Pombiliti contains a working version of this enzyme that’s administered into the bloodstream, while Opfolda is an oral medication that helps to stabilize the enzyme so it can function better.

Full approval in the EU for adults with late-onset Pompe disease

The two-part therapy recently earned full approval to treat adults with LOPD in the European Union. It’s also up for potential approval in the U.S., with a decision expected before the end of the year.

Approvals of Pombiliti plus Opfolda in the U.K. and EU were supported by data from the Phase 3 clinical trial PROPEL (NCT03729362). The study compared the combination therapy against alglucosidase alfa, an approved enzyme replacement therapy marketed under the name Lumizyme in the U.S., and Myozyme elsewhere.

Findings from PROPEL showed that Pombiliti plus Opfolda led to significant improvements in patients’ lung function and exercise abilities. Results with the combination therapy tended to be slightly better than with Lumizyme/Myozyme, though some differences did not reach statistical significance.

“We are grateful to the global Pompe community who have helped advance this therapy, especially the patients, families, and physicians who participated in our clinical studies,” Campbell said.

Amicus is currently sponsoring a Phase 3 trial called ROSSELLA (NCT04808505) testing Pombiliti plus Opfolda in children with infantile-onset Pompe disease ages 6 months to 17 years. Recruitment is ongoing at two U.S. sites in Georgia and Florida.