Related Experiment Video
Updated: Jul 28, 2025
08:36
Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-3
Published on: April 7, 2023
1.1K
Leniolisib: First Approval
Sean Duggan1, Zaina T Al-Salama2
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.
Drugs
|May 31, 2023
Summary
Leniolisib, a selective phosphoinositide 3-kinase-delta inhibitor, is now approved for activated PI3Kδ syndrome (APDS). This marks a significant milestone for treating this rare immunodeficiency disorder.
Area of Science:
- Immunology
- Pharmacology
- Genetics
Background:
- Activated PI3Kδ syndrome (APDS) is a rare primary immunodeficiency disorder.
- Phosphoinositide 3-kinase-delta (PI3Kδ) pathway dysregulation is central to APDS pathogenesis.
- Targeting the PI3Kδ pathway offers a potential therapeutic strategy for APDS.
Purpose of the Study:
- To summarize the development milestones of leniolisib.
- To highlight the regulatory journey leading to the first approval of leniolisib for APDS.
- To provide an overview of leniolisib as a PI3Kδ inhibitor for immunodeficiency disorders.
Main Methods:
- Review of clinical development data and regulatory submissions for leniolisib.
- Analysis of scientific literature on PI3Kδ inhibitors and APDS.
- Summary of key preclinical and clinical trial findings (details not provided in abstract).
Main Results:
- Leniolisib (JOENJA®) received its first approval in March 2023 for APDS in patients 12 years and older.
- Leniolisib is under regulatory review in the European Union for APDS.
- Development for Sjögren's syndrome was discontinued.
Conclusions:
- Leniolisib represents a novel targeted therapy for APDS.
- The approval signifies a breakthrough in treating this specific immunodeficiency.
- Further regulatory reviews are ongoing in other regions.
More Related Videos
Related Concept Videos
01:21Drugs for Treatment of Constipation-Predominant IBS
236
Pharmacological therapies for IBS-C are designed to alleviate abdominal discomfort and enhance bowel function. In patients with IBS-C, fiber supplements may help soften stools and decrease straining, but may also lead to increased gas production and bloating. Osmotic laxatives like milk of magnesia are frequently used to soften stools and increase stool frequency in IBS-C patients. In addition, two drugs approved for use in severe IBS-C adult cases are linaclotide (Linzess) and lubiprostone...
236
01:02Prescription, Nonprescription and Orphan Drugs
791
Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
791
01:26Drug Administration and Therapy Phases: Overview
564
Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
564
01:29Drugs for Treatment of Ulcerative Colitis in IBD
181
Ulcerative colitis is a chronic inflammatory condition primarily affecting the colon and rectum. The primary drugs used in the treatment of ulcerative colitis are aminosalicylates. They exhibit anti-inflammatory and immunosuppressive properties. They modulate inflammatory mediators and inhibit the activity of nuclear factor κB (NF-κB). Aminosalicylates also reduce inflammation by inhibiting prostaglandin and leukotriene production and decreasing neutrophil chemotaxis and superoxide...
181
01:17Drugs for Treatment of Diarrhea-Predominant IBS
223
Diarrhea-predominant irritable bowel syndrome (IBS-D) is a subtype of IBS characterized primarily by frequent, loose, or watery stools, abdominal pain, and abdominal discomfort. Therapeutic approaches to managing IBS-D include dietary changes, stress management techniques, and pharmaceutical interventions.
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
223
01:25Drug Regulation
1.4K
Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
1.4K