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Practical NeurologyPombiliti + Opfolda Approved by FDA as Combination Therapy for Late-Onset Pompe Disease

 New Combination Therapy Offers Hope for Patients Not Improving on Current Treatments

The Food and Drug Administration (FDA) has given the green light to a new combination therapy for late-onset Pompe disease (LOPD). This approval marks a significant step forward in the treatment of LOPD, offering a new option for patients who are not responding to current enzyme replacement therapies.

HCN Medical Memo
This FDA approval represents a significant advancement in the treatment landscape for LOPD. For healthcare professionals, it’s crucial to stay updated on these developments to provide the best possible care for patients. This new combination therapy could potentially improve patient outcomes and quality of life.

Key Points

  • The FDA has approved Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) combination therapy for people with LOPD who weigh at least 40 kg and are not improving on current enzyme replacement therapy (ERT).
  • The approval is based on the results of the PROPEL phase 3 double-blind clinical trial that enrolled 125 adult participants with diagnosed LOPD.
  • Treatment with Pombiliti + Opfolda resulted in an improvement of 14 m in 6-minute walk distance (6MWD) and a 3% improvement in sitting forced vital capacity (FVC).
  • Dr. Tahseen Mozaffar, an investigator for the PROPEL study, highlighted the importance of this new treatment option for adults living with LOPD and not improving on current therapies.

Pompe disease is an inherited lysosomal disorder caused by deficiency of the acid alpha-glucosidase (GAA) enzyme, leading to glycogen accumulation within cells.

Additional Points

  • Pombiliti + Opfolda therapy should be started 2 weeks after the last ERT dose.
  • Pombiliti’s label contains a boxed warning for hypersensitivity reactions including anaphylaxis, infusion-associated reactions, and risk of acute cardiorespiratory failure in susceptible patients.

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