Joenja

Joenja is a prescription medicine used to treat a rare, genetic disease that affects the immune system called activated phosphoinositide 3-kinase delta syndrome (APDS).

APDS is a type of primary immunodeficiency disorder that causes increased susceptibility to infections.

Joenja contains leniolisib, which belongs to a class of medicines called phosphoinositide 3-kinase delta (PI3Kδ) inhibitors. Leniolisib blocks PI3Kδ, an enzyme that is overactive in people with APDS. Blocking it helps correct the signaling that interferes with the normal development and function of immune cells.

Joenja is used to treat activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and children 4 years of age and older who weigh 27 kg (about 59.5 pounds) or more.

APDS is a primary immunodeficiency disease caused by genetic mutations in either the PIK3CD or PIK3R1 genes, which leads to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway. Balanced signaling in the PI3Kδ pathway is essential for normal development and function of immune cells in the body.

Symptoms of APDS include frequent and severe infections of the ears, sinuses, and upper and lower respiratory tracts, as well as autoimmunity and inflammatory symptoms. People with APDS may also be at higher risk for cancers such as lymphoma.

Joenja has not been studied in children younger than 4 years of age or in children who weigh less than 27 kg.

Joenja can cause harm to an unborn baby. Tell your healthcare provider if you are pregnant or plan to become pregnant.

If you are able to become pregnant, you will need to use highly effective birth control during treatment and for one week after the last dose.

Joenja can cause serious allergic reactions, including anaphylaxis. Stop taking Joenja and get medical help straight away if you develop signs of a serious allergic reaction, such as rash, hives, swelling of your face, lips, tongue or throat, trouble breathing, or dizziness or fainting.

Live vaccines may not work as well while you are taking Joenja. Tell your healthcare provider that you take Joenja before you have any vaccination.

Before you start treatment, tell your healthcare provider about all of your medical conditions, including if you:

• have liver problems. Joenja is not recommended if you have moderate to severe liver impairment
• have ever had a serious allergic reaction to Joenja or any of its ingredients
• are due to have a vaccination
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed

Pregnancy

Tell your healthcare provider if you are pregnant or plan to become pregnant. Joenja may harm your unborn baby. Your healthcare provider will do a pregnancy test before you start treatment.

Use highly effective birth control to prevent pregnancy during treatment, and for one week after you stop treatment. Talk to your doctor about what type of birth control method is right for you.

Breastfeeding

It is not known if this medicine passes into your breast milk. Breastfeeding is not advised during treatment, and for one week after the last dose.

Talk to your doctor about the best way to feed your baby during treatment.

The most common side effects in adults and children 12 years of age and older are:

• headache
• inflammation of the sinuses (sinusitis)
• atopic dermatitis or eczema (dry, itchy, and inflamed skin)

Children 4 years to less than 12 years of age also commonly reported abdominal pain, cough, and respiratory tract infection.

Other side effects reported during the placebo-controlled study included fast heartbeat, diarrhea, tiredness, fever, back pain, neck pain, and hair loss.

Some people taking Joenja had a fall in one type of white blood cell (neutrophils), though no infections linked to this were reported. Weight gain has also been reported, and some people became overweight or obese.

Serious side effects

Serious allergic reactions, including anaphylaxis, have been reported since Joenja was approved. Stop taking Joenja and get emergency medical help if you have signs of an allergic reaction such as hives, rash, swelling of your face, lips, tongue, or throat, difficulty breathing, or feeling faint.

These are not all the possible side effects. Tell your healthcare provider about any side effect that bothers you or does not go away.

• Take Joenja tablets twice daily, approximately 12 hours apart.
• The tablets can be taken with or without food.
• If you miss a dose by more than 6 hours, skip that dose and take the next dose at the usual time.
• If you vomit within 1 hour after taking your dose, take another dose as soon as possible. If vomiting occurs more than 1 hour after dosing, wait and take the next dose at the usual time.
• Store tablets at 68°F to 77°F (20°C to 25°C). Brief excursions between 59°F and 86°F (15°C to 30°C) are permitted. Do not refrigerate.

Dosing information

The dose of Joenja depends on age and body weight.

Joenja is available as 40 mg, 50 mg, and 70 mg film-coated tablets.

Pregnancy status is checked in females of reproductive potential before treatment starts.

Joenja is not recommended for people with moderate to severe liver impairment.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Joenja and certain other medicines may affect each other.

Medicines that should be avoided during treatment with Joenja include:

• strong CYP3A4 inhibitors, which can raise the amount of leniolisib in your body
• strong and moderate CYP3A4 inducers, which can lower the amount of leniolisib in your body and make it work less well
• medicines carried by the BCRP, OATP1B1, or OATP1B3 transporters, such as rosuvastatin. Joenja raises the levels of these medicines, which may increase the risk of their side effects

Joenja can be taken with medicines that reduce stomach acid, such as proton pump inhibitors and H2 blockers.

Combined birth control pills containing ethinyl estradiol and levonorgestrel are still expected to work during treatment with Joenja.

Tell your healthcare provider if you are scheduled to receive an immunization (vaccine). Live, weakened vaccines may be less effective if they are given during treatment with Joenja.

Know the medicines you take. Keep a list of your medicines and show it to your healthcare provider and pharmacist when you get a new medicine.

Active ingredient: leniolisib phosphate

Inactive ingredients: colloidal silicon dioxide, hydroxypropyl methylcellulose, lactose monohydrate, magnesium stearate, microcrystalline cellulose, and sodium starch glycolate. The tablet film coating contains hydroxypropyl methylcellulose, iron oxide black (50 mg tablets), iron oxide red (40 mg, 50 mg, and 70 mg tablets), iron oxide yellow (70 mg tablets), polyethylene glycol, talc, and titanium dioxide.

Manufactured for: Pharming Healthcare Inc., 10 Independence Blvd., Warren, NJ 07059 USA. To report suspected side effects, call 1-800-930-5221, or contact the FDA at 1-800-FDA-1088.