The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Amicus Therapeutics’ combination treatment of Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat) for adult patients with late-onset Pompe disease.

Pompe disease is a rare genetic disorder caused by the deficiency of alpha-glucosidase, which helps in glycogen breakdown, leading to build-up of glycogen in muscles, causing weakness. As per the National Organisation for Rare Diseases, 1 in 40,000 infants born in the US have Pompe disease.

Pombiliti has been approved as a long-term enzyme replacement therapy in combination with miglustat, a glucosylceramide synthase enzyme inhibitor. Amicus’ miglustat drug was approved by the European Commission in June under the brand name Opfolda. Janssen also markets a miglustat drug with the brand name Zavesca to treat Gaucher’s disease.

Pompe disease is currently treated with enzyme replacement therapy, namely Sanofi’s Myozyme (alglucosidase alfa), and therapies addressing specific symptoms.

The drugs received approval through the Innovation Passport under the Innovative Licensing and Access Pathway. Furthermore, the drug combination was granted an orphan designation allowing for market exclusivity in the UK for ten years. Earlier this year, the Pombiliti and Opfolda combination was approved by the European Commission for marketing in the EU.

The UK National Institute for Health and Care Excellence (Nice) has also recommended the Pombiliti and Opfolda combinations as the first and later-line therapy for late-onset Pompe disease in adults, respectively.

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The MHRA approval and NICE recommendation were based on Phase III study (NCT03729362) data showing the clinical improvement with Amicus drugs compared with standard Myozyme treatment.

As per GlobalData, the market for Pompe disease is expected to grow to $1.95bn by 2030, mostly due to the introduction of novel therapies and increased prevalence of the disease. Myozyme generated $208m in sales in Q2 2023, as per Sanofi’s Q2 earnings report.

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