FDA Approves Joenja As The First Treatment For APDS

Introduction

In the ever-evolving landscape of medical advancements, Joenja has emerged as a notable player, offering potential therapeutic benefits for a specific group of individuals. Understanding its nuances, from FDA approval to indications and precautions, is crucial for both healthcare professionals and patients.

Joenja (leniolisib), a novel oral medication, has emerged as a beacon of hope for patients with APDS. Joenja received FDA approval in 2023 for the treatment of adult and pediatric patients with APDS. This significant milestone marked the first approved therapy specifically targeting APDS, providing a much-needed treatment option for patients and their families.

FDA Approval and Manufacturing Details

On March 24, 2023, the FDA approved Joenja for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) which is manufactured by the company called Skyepharma Production SAS. 

Indication 

Joenja is a kinase inhibitor approved for addressing activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in individuals aged 12 years and older, both in adults and pediatric patients.

APDS is caused by mutations in the PIK3CD gene, which encodes the p110δ subunit of the phosphoinositide 3-kinase delta (PI3Kδ) enzyme. This enzyme plays a critical role in regulating the immune system. Mutations in the PIK3CD gene lead to an overactivation of PI3Kδ, resulting in the overactivity of the immune system.

Mechanism Of Action

Joenja works by targeting PI3Kδ, specifically inhibiting its overactive activity. By blocking PI3Kδ, Joenja helps to restore normal immune function and reduce the symptoms of APDS.

What is APDS?

Activated phosphoinositide 3-kinase delta syndrome (APDS) is a rare genetic immunodeficiency disorder caused by mutations in the phosphoinositide 3-kinase delta (PI3Kδ) gene. This condition affects the immune system, leading to recurrent infections, impaired antibody production, and immune dysregulation.

Symptoms of Activated phosphoinositide 3-kinase delta syndrome (APDS) include:

1. Recurrent infections (respiratory, ear, sinus infections)
2. Immune dysregulation leading to autoimmune issues
3. Enlarged lymph nodes and spleen
4. Chronic lung problems like bronchiectasis
5. Allergies and allergic reactions

Dosage Information and Drug Interactions

Joenja tablet is administered orally as tablets, with or without food. The recommended dosage for adults and pediatric patients 12 years of age and older weighing 45 kg or greater is 70 mg administered twice daily approximately 12 hours apart.

Joenja has the potential to interact with certain medications, including

•  CYP3A4 inhibitors (medications that can increase the blood levels of Joenja)
•  CYP3A4 inducers (medications that can decrease the blood levels of Joenja)

It is crucial to inform your doctor about all medications you are taking, including prescription and over-the-counter medications, herbal supplements, and vitamins, before starting Joenja.

Mode of Administration

Joenja is administered orally as tablets, providing a convenient and easy-to-use treatment option for patients. The tablets should be swallowed whole with water, and patients should avoid crushing, chewing, or splitting the tablets.

Usage in Specific Populations

Pregnancy: Joenja may harm the fetus, based on animal studies. Limited data on its use in pregnant women hinder a full assessment of the risk of birth defects, miscarriage, or other adverse outcomes for both mother and fetus.

Lactation: Joenja poses fetal harm based on animal studies. Limited data on pregnant women hinder a comprehensive assessment of birth defects, miscarriages, or adverse outcomes risks for both mother and fetus.

Contraception: Women of reproductive potential are recommended to use highly effective contraception while undergoing Joenja treatment and to continue contraception for one week after completing the course.

Pediatric Use: Joenja’s safety and efficacy in treating activated phosphoinositide 3-kinase delta syndrome have been established in pediatric patients aged 12 years and older.

Geriatric Use: Since clinical studies on Joenja did not encompass patients aged 65 and older, any potential differences in response compared to younger adults remain undetermined.

Hepatic Impairment: Leniolisib undergoes extensive (60%) hepatic metabolism. The impact of hepatic impairment on leniolisib’s pharmacokinetics has not been investigated. The use of Joenja is not recommended in patients with moderate to severe hepatic impairment.

Side Effects

The most common side effects associated with Joenja include:

•  Diarrhea
•  Upper respiratory tract infections
•  Headache
•  Fatigue
•  Nausea
•  Vomiting

These side effects are usually mild and manageable. However, if you experience any severe or persistent side effects, contact your doctor immediately.

Conclusion

Joenja represents a significant advancement in the treatment of APDS, offering a targeted and effective approach that specifically addresses the underlying genetic cause of the disease. Its FDA approval marks a turning point in the management of APDS, providing a much-needed treatment option for patients and their families. With continued research and clinical experience, Joenja has the potential to further enhance APDS management and improve patient outcomes.

FAQs

Who can take Joenja?

Joenja is approved for use in adult and pediatric patients 12 years of age and older with APDS.

What is the dosage of Joenja?

The recommended dosage of Joenja for adults and pediatric patients 12 years of age and older weighing 45 kg or greater is 70 mg administered twice daily approximately 12 hours apart.

How is Joenja taken?

Joenja is taken orally as tablets, with or without food. The tablets should be swallowed whole with water, and patients should avoid crushing, chewing, or splitting the tablets.

Are there any drug interactions with Joenja®?

Joenja has the potential to interact with certain medications, including CYP3A4 inhibitors and CYP3A4 inducers. It is crucial to inform your doctor about all medications you are taking, including prescription and over-the-counter medications, herbal supplements, and vitamins, before starting Joenja.

How to buy Joenja online?

Acquiring Joenja online has become more convenient. To obtain this medication, exclusive to the US and Europe, you can reach out to the SANSFRO team or other specialized firms experienced in importing medicines from these regions.

What is Joenja price?

Joenja price depends on various product specifications and factors. For precise pricing information, please get in touch with our Patient Support Team at ‎(+91) 93157 05373 or email us at help@sansfro.com. Our team can provide you with accurate and current cost details for Joenja in the Indian market.

Reference:

1. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217759s000lbl.pdf
2. https://www.drugs.com/joenja.html