Complete adverse effect profile including incidence rates and management
Important Safety Information
This is not a complete list of all possible side effects. Contact your healthcare provider if you experience any unexpected symptoms. For serious or life-threatening side effects, seek emergency medical attention immediately.
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Infusion-Associated Reactions [see Warnings and Precautions (5.2) ] Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain and pyrexia.
( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
Adverse Reactions from the Pooled Clinical Trials Including Trial 1 The pooled safety analysis from 3 clinical trials included 151 adult patients with late-onset Pompe disease (LOPD) treated with POMBILITI in combination with Opfolda including: 85 patients in the randomized, double-blind, active-controlled trial in adults (Trial 1) [see Clinical Studies (14) ] , 37 patients in the open-label extension trial where patients switched from a non‑U.S.‑approved alglucosidase alfa product [see Clinical Studies (14) ] to POMBILITI in combination with Opfolda, 29 patients in an open-label trial.
The total median duration of exposure in these trials was 21 months, with 120 patients having at least 12 months exposure to POMBILITI in combination with Opfolda.
In these trials, 78% (n=117) of the patients received previous ERT (ERT‑experienced) with a mean treatment duration of 7.7 years.
In these trials, 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 in these trials permanently discontinued POMBILITI due to adverse reactions, including 4 of these patients who discontinued the treatment because of a serious adverse reaction.
The most common adverse reactions (≥5%) reported in the pooled safety population of patients treated with POMBILITI in combination with Opfolda in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain and pyrexia.
In these trials, IARs were reported in 48 (32%) patients treated with POMBILITI in combination with Opfolda.
5 WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity: May cause embryo-fetal harm.
Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose.
( 4 , 5.4 , 8.1 , 8.3 ) Risks Associated with Opfolda: Refer to the Opfolda Prescribing Information for a description of additional risks for Opfolda.
( 5.5 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in POMBILITI-treated patients.
In clinical trials, 43 (27%) POMBILITI-treated patients experienced hypersensitivity reactions, including 4 (3%) patients who reported severe hypersensitivity reactions and 4 (3%) additional patients who experienced anaphylaxis (fulfilling at least one of the Sampson criteria).
Like all medications, Pombiliti Atga can cause side effects. However, not everyone who takes this medication will experience them. Many side effects are dose-dependent and may improve as your body adjusts to the medication. Others may require dose adjustment or medical attention.
Contact your healthcare provider promptly if you experience:
Seek immediate emergency medical care if you experience signs of: