JOENJA

The systemic drug exposure (AUC and C max ) of leniolisib increased dose proportionally within the studied range of doses (20 to 140 mg twice a day dosing and single doses of 10 to 400 mg). During twice daily dosing approximately 12 hours apart, leniolisib accumulates approximately 1.4-fold (range of 1.0 to 2.2) in achieving steady-state, consistent with an effective half-life (t 1/2 ) of approximately 7 hours. Steady state drug concentrations can be expected to be reached after approximately 2 ...

The efficacy of JOENJA was evaluated in the placebo-controlled portion of

Study 2201(NCT02435173), a 12-week blinded, randomized, placebo-controlled study in adult and pediatric patients 12 years of age and older with confirmed APDS-associated genetic PI3Kδ mutation with a documented variant in either PIK3CD or PIK3R1. Baseline patient demographics are shown in Table 2.

Table 2Baseline Demographic and Disease Characteristics in Patients with APDS (Study 2201) Demographics and Disease Characteristics JOENJA (N=21) Placebo (N=10) Demographics 1 Patient age from study Day -4 up to initial JOENJ...

The following clinically significant adverse reactions are described elsewhere in the labeling: Risk of Hypersensitivity Reactions, Including Anaphylaxis [see Warnings and Precautions (5.3) ] Most common adverse reactions (incidence > 10%) were headache, sinusitis, and atopic dermatitis. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Pharming Healthcare Inc. at 1-800-930-5221 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are cond...

None. None. ( 4 )

Embryo-Fetal Toxicity: JOENJA may cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception. ( 5.1 , 8.1 , 8.3 ) Vaccinations: Live, attenuated vaccinations may be less effective if administered during JOENJA treatment. ( 5.2 ) Risk of Hypersensitivty Reactions Including Anaphylaxis: Hypersensitivity reactions including anaphlyaxis have been reported in the postmarketing setting. If hypersensitivity reactions occur, discontinue JOENJA and institute app...

7 DRUG INTERACTIONS Strong CYP3A4 Inhibitors: Avoid concomitant use. ( 7.1 ) Strong and Moderate CYP3A4 Inducers: Avoid concomitant use. ( 7.1 ) BCRP, OATP1B1, and OATP1B3 Substrates: Avoid concomitant use. ( 7.2 ) 7.1 Effects of Other Drugs on JOENJA Strong CYP3A4 Inhibitors Concomitant use of JOENJA with strong CYP3A4 inhibitors should be avoided. JOENJA is a substrate of CYP3A4. Leniolisib expo...