Agamree Side Effects

6 ADVERSE REACTIONS The following serious adverse reactions are discussed in more detail in other sections: Alterations in Endocrine Function [see Warnings and Precautions ( 5.1 )] Immunosuppression and Increased Risk of Infection [see Warnings and Precautions ( 5.2 )] Alterations in Cardiovascular/Renal Function [see Warnings and Precautions ( 5.3 )] Gastrointestinal Perforation [see Warnings and Precautions ( 5.4 )] Behavioral and Mood Disturbances [see Warnings and Precautions ( 5.5 )] Effects on Bones [see Warnings and Precautions ( 5.6 )] Ophthalmic Effects [see Warnings and Precautions ( 5.7 )] Immunizations [see Warnings and Precautions ( 5.8 )] Effects on Growth and Development [see Warnings and Precautions ( 5.9 )] Myopathy [see Warnings and Precautions ( 5.10 )] Kaposi's Sarcoma [see Warnings and Precautions ( 5.11 )] Thromboembolic Events [see Warnings and Precautions ( 5.12 )] Anaphylaxis [see Warnings and Precautions ( 5.13 )] The most common adverse reactions (>10% for AGAMREE and greater than placebo) are cushingoid features, psychiatric disorders, vomiting, weight increased, and vitamin D deficiency.

( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Catalyst Pharmaceuticals, Inc.

at 1-844-347-3277 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.

Common Adverse Reactions in Clinical Studies Table 1 lists the adverse reactions that occurred in ≥ 5% of the patients treated with AGAMREE 6 mg/kg/day (N=28) or AGAMREE 2 mg/kg/day (N=30) and that occurred more frequently than in the patients who received placebo (N=29) in Study 1 [see Clinical Studies ( 14 )] , which was 24 weeks and included patients with DMD between the ages of 4 and 7 years.

Table 1: Adverse Reactions in Patients with DMD that Occurred in ≥ 5% of Patients Treated with AGAMREE and More Frequently than in Patients Who Received Placebo During 24 Weeks (Study 1) 1 Includes the following adverse reactions that occurred more frequently in the AGAMREE group than in placebo: abnormal behavior, aggression, agitation, anxiety, irritability, mood altered, sleep disorder, and stereotypy.

Adverse Reaction AGAMREE 2 mg/kg/d (N=30) % AGAMREE 6 mg/kg/d (N=28) % Placebo (N=29) % Cushingoid Features 7 29 0 Psychiatric disorders 1 7 21 14 Vomiting 17 14 7 Weight increased 0 11 3 Vitamin D deficiency 7 11 0 Cough 10 7 3 Headache 7 7 3 Diarrhea 3 7 3 Increased appetite 3 7 3 Rhinitis 3 7 3 In a separate open-label safety study of pediatric patients aged 2 to less than 4 years (n=16) and pediatric patients aged 7 to less than 18 years (n=16) with DMD, adverse reactions were similar to those seen in the Study 1 pediatric patients.