A two-component therapy for adults with late-onset Pompe disease (LOPD) weighing 88 lbs or more who are not improving on their current enzyme replacement therapy (ERT).
Pombiliti+Opfoldaresetting expectations
POMBILITI + OPFOLDA was shown to improve walking distance and breathing function vs the comparator* in ERT-experienced adults
Study Design
POMBILITI + OPFOLDA was evaluated in a randomized, controlled clinical study over 52 weeks. This was the largest global, phase 3 study ever conducted in LOPD.
123 PEOPLE TOTAL IN THE STUDY, INCLUDING:
ERT-experienced
People in this group were on an ERT for at least 2 years
ERT-naïve†
People in this group had never received an ERT
85 people received POMBILITI + OPFOLDA
38 people received the comparator
- The study’s primary endpoint (goal) was to see how much farther people who received POMBILITI + OPFOLDA could walk in 6 minutes, using the 6-Minute Walk Test (6MWT)‡
- Forced vital capacity (FVC), a test of breathing ability, was a key secondary endpoint (goal) of the study
- ERT-experienced people were on treatment for an average of 7.4 years before the study
*An alglucosidase alfa product not approved in the US + placebo.
†POMBILITI + OPFOLDA is not approved for use in ERT-naïve people.
‡Results of the 6MWT were numerically favorable but did not meet the primary endpoint of statistical superiority for the full study population.
Results for ERT-experienced adults
Improvements inWalking Distance
Study participants were assessed using a test called the 6-Minute Walk Test (6MWT), a timed test to measure how far a person can walk in 6 minutes.
ERT-experienced adults receiving POMBILITI + OPFOLDA had improved walking distance versus adults receiving the comparator§
- People who received POMBILITI + OPFOLDA walked an average of 53.5 feet farther from the start of the study
- People who received the comparator walked an average of 2.3 feet farther from the start of the study
- This indicated an estimated treatment effect of more than 50 feet for POMBILITI + OPFOLDA
§Results of the 6MWT and FVC for ERT-experienced people were numerically favorable but were not tested to determine statistical superiority of POMBILITI + OPFOLDA vs the comparator.
Improvements inBreathing
Study participants were assessed using forced vital capacity (FVC), a test of breathing ability.
ERT-experienced adults receiving POMBILITI + OPFOLDA had improved breathing function versus adults receiving the comparator§
- People who received POMBILITI + OPFOLDA who had previous experience with ERT saw their breathing increase by an average of 0.1% from the start of the study
- ERT-experienced adults who received the comparator saw their breathing decline by an average of 3.5% from the start of the study
- This indicated an estimated treatment effect of 3.5% for POMBILITI + OPFOLDA
§Results of the 6MWT and FVC for ERT-experienced people were numerically favorable but were not tested to determine statistical superiority of POMBILITI + OPFOLDA vs the comparator.
DataHex4 Measurements
Study participants were tested for Hex4, a measure of glycogen buildup
What is the Hex4 test?
It is a measure of glycogen accumulation.
- Lower Hex4 test results are a sign that there is less glycogen accumulation
- People receiving POMBILITI + OPFOLDA who had previous experience with ERT saw an average reduction of 29% in Hex4 vs an average increase of 20% with the comparator
Safety
Information about side effects with POMBILITI + OPFOLDA
The most common side effects (≥5%) reported during the phase 3 PROPEL study were headache and diarrhea.
The most common side effects reported in at least 5% of patients treated with POMBILITI + OPFOLDA across 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain, and fever.
Serious adverse reactions reported in 2 or more patients treated with POMBILITI in combination with OPFOLDA were anaphylaxis and urticaria. A total of 5 patients treated with POMBILITI in combination with OPFOLDA permanently discontinued POMBILITI due to adverse reactions, including 4 of these patients who discontinued the treatment because of a serious adverse reaction.
Additional side effects
Side effects reported in at least 2% of adults treated with POMBILITI + OPFOLDA across 3 clinical trials included: muscle aches, joint pain, increased blood pressure, pain, tremor (shaking), indigestion, weakness; constipation, infusion site swelling, flank (side) pain, feeling unwell, burning or pins and needles feeling, and decreased platelet count.
POMBILITI in combination with OPFOLDA may cause serious side effects, including: hypersensitivity reactions (including anaphylaxis), infusion-associated reactions, and risk of acute cardiorespiratory failure. Please see IMPORTANT SAFETY INFORMATION.
These are not all of the possible side effects of POMBILITI in combination with OPFOLDA.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Discover the dosing schedule
See how POMBILITI + OPFOLDA is administered.
Important Safety Information
POMBILITI in combination with OPFOLDA may cause serious side effects, including:
- Hypersensitivity reactions (including Anaphylaxis): Severe and potentially life-threatening allergic-type reactions related to the infusion may occur during and after POMBILITI in combination with OPFOLDA treatment. Your doctor will inform you of the signs and symptoms of hypersensitivity reactions which may include: difficulty breathing or swallowing, rash or hives, low blood pressure, swelling of lips, tongue, throat or face. Seek medical care immediately should signs and symptoms occur. If a severe reaction occurs, your doctor may decide to immediately discontinue the infusion and provide medical care. Appropriate medical support measures may be administered, and you may require close observation during and after POMBILITI infusion.
What are POMBILITI and OPFOLDA?
POMBILITI and OPFOLDA are prescription medicines used in combination for the treatment of adults with late-onset Pompe disease weighing 88 pounds (40 kg) or more and who are not improving on their current enzyme replacement therapy (ERT).
It is not known if POMBILITI in combination with OPFOLDA is safe and effective in children with late-onset Pompe disease.
Important safety information
POMBILITI in combination with OPFOLDA may cause serious side effects, including:
- Hypersensitivity reactions (including Anaphylaxis): Severe and potentially life-threatening allergic-type reactions related to the infusion have been reported during and after POMBILITI in combination with OPFOLDA treatment. Your doctor will inform you of the signs and symptoms of hypersensitivity reactions which may include: difficulty breathing or swallowing; rash or hives; low blood pressure; swelling of lips, tongue, throat, or face. Seek medical care immediately should signs and symptoms occur. If a severe reaction occurs, your doctor may decide to immediately discontinue the infusion and provide medical care. Appropriate medical support measures may be administered, and you may require close observation during and after POMBILITI infusion.
- Infusion-Associated Reactions (IARs): Severe IARs related to the infusion have been reported during or after POMBILITI in combination with OPFOLDA. Your doctor will inform you of the signs and symptoms of hypersensitivity reactions which may include: hives, itching, shortness of breath, flushing, chills, and low blood pressure. Seek medical care immediately should signs and symptoms occur. If severe IARs occur during infusion, your doctor may decide to immediately discontinue the infusion and provide appropriate medical care. If you have an acute underlying illness at the time of POMBILITI infusion you may be at greater risk for IARs. If you have advanced Pompe disease you may have compromised heart and breathing function, which may put you at a higher risk of severe complications from IARs.
- Risk of Acute Cardiorespiratory Failure: If you are likely to develop fluid volume overload or have an acute breathing condition or heart and/or breathing problems that require fluid restriction, you may be at risk of worsening of your heart or breathing status during POMBILITI infusion. Your doctor may decide that close observation during and after POMBILITI administration may be necessary.
Do not take POMBILITI in combination with OPFOLDA if you are pregnant.
Before taking POMBILITI in combination with OPFOLDA, tell your healthcare provider about all of your medical conditions, including if you:
- have kidney problems
- are pregnant or plan to become pregnant. POMBILITI in combination with OPFOLDA may cause harm to your unborn baby.Females who are able to become pregnant:
- Your healthcare provider will check if you are pregnant before you start treatment with POMBILITI in combination with OPFOLDA.
- You should use effective birth control (contraception) during treatment with POMBILITI in combination with OPFOLDA and for at least 60 days after the last dose.
- Tell your healthcare provider right away if you become pregnant or think you might be pregnant during treatment with POMBILITI in combination with OPFOLDA.
- are breastfeeding or plan to breastfeed. It is not known if OPFOLDA alone or in combination with POMBILITI passes into your breast milk. Do not breastfeed during treatment with POMBILITI in combination with OPFOLDA. Talk to your healthcare provider about the best way to feed your baby during this time.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
POMBILITI and OPFOLDA must be taken in combination. POMBILITI in combination with OPFOLDA will be given to you 1 time every other week.
The most common side effects of POMBILITI in combination with OPFOLDA include: headache, diarrhea, tiredness, nausea, stomach area pain, and fever.
POMBILITI in combination with OPFOLDA may cause fertility problems in females and males, which may affect the ability to have children. Talk to your healthcare provider if you have concerns about fertility.
These are not all the possible side effects of POMBILITI and OPFOLDA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
What are POMBILITI and OPFOLDA?
POMBILITI and OPFOLDA are prescription medicines used in combination for the treatment of adults with late-onset Pompe disease weighing 88 pounds (40 kg) or more and who are not improving on their current enzyme replacement therapy (ERT).
It is not known if POMBILITI in combination with OPFOLDA is safe and effective in children with late-onset Pompe disease.
Please see full Prescribing Information, including BOXED WARNING, for POMBILITI and full Prescribing Information and Patient Information for OPFOLDA.