Global BulletinAll NewsFDA Approval AlertWomen in Oncology
Expert InterviewsAround the PracticeBetween the LinesFace OffFrom All AnglesMeeting of the MindsOncViewPodcastsTraining AcademyTreatment Algorithms with the Oncology Brothers
Conferences
All JournalsEditorial BoardFor AuthorsYear in Review
Frontline ForumSatellite Sessions
CME/CE
Awareness MonthInteractive ToolsNurse Practitioners/Physician's AssistantsPartnersSponsoredSponsored Media
Career CenterSubscribe
Adverse Effects
Brain Cancer
Breast CancerBreast CancerBreast Cancer
Gastrointestinal CancerGastrointestinal CancerGastrointestinal CancerGastrointestinal CancerGastrointestinal CancerGastrointestinal Cancer
Genitourinary CancersGenitourinary CancersGenitourinary CancersGenitourinary Cancers
Gynecologic CancersGynecologic CancersGynecologic CancersGynecologic Cancers
Head & Neck Cancer
Hematologic OncologyHematologic OncologyHematologic OncologyHematologic Oncology
InfectionInfection
Leukemia
Lung CancerLung CancerLung Cancer
Lymphoma
Neuroendocrine Tumors
Oncology
Pediatric Cancers
Radiation Oncology
Sarcoma
Screening
Skin Cancer & Melanoma
Surgery
Thyroid Cancer
Spotlight -
  • Radiation Oncology
  • Surgery
Adverse Effects
Brain Cancer
Breast CancerBreast CancerBreast Cancer
Gastrointestinal CancerGastrointestinal CancerGastrointestinal CancerGastrointestinal CancerGastrointestinal CancerGastrointestinal Cancer
Genitourinary CancersGenitourinary CancersGenitourinary CancersGenitourinary Cancers
Gynecologic CancersGynecologic CancersGynecologic CancersGynecologic Cancers
Head & Neck Cancer
Hematologic OncologyHematologic OncologyHematologic OncologyHematologic Oncology
InfectionInfection
Leukemia
Lung CancerLung CancerLung Cancer
Lymphoma
Neuroendocrine Tumors
Oncology
Pediatric Cancers
Radiation Oncology
Sarcoma
Screening
Skin Cancer & Melanoma
Surgery
Thyroid Cancer
    • Conferences
    • CME/CE
    • Career Center
    • Subscribe
Advertisement

FDA Grants Accelerated Approval to Alpelisib in PIK3CA-Related Overgrowth Spectrum

April 9, 2022
By Ariana Pelosci
Article

Based on results from the EPIK-P1 trial, the FDA has granted accelerated approval to alpelisib for PIK3CA-related overgrowth spectrum.

The FDA has granted accelerated approval to alepelisib (Vijoice) for adult and pediatric patients 2 years or older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy, according to a press release from the FDA.1 

The approval is based off the results of EPIK-P1 (NCT04285723), a single-arm, retrospective chart review of patients who were 2 or older with PROS who were treated with alepelisib. The drug was administered as part of an expanded access program for compassionate use.

PROS is a group of rare disorders that cause overgrowth in the body due to the mutations in the PIK3CA gene.2 Disorders associated with PROS include fibroadipose hyperplasia, CLOVES syndrome, and Megalencephaly-capillary malformation syndrome. Signs and symptoms of PROS include having a larger-than normal brain, low muscle tone, seizures, intellectual disability, and changes in the blood vessels. Since cells are likely to grow and divide more quickly in this population, those with PROs are at an increased risk of developing cancer. Colon, breast, brain, liver, stomach, and lung cancer are the most likely to have PIK3CA mutations among those without PROS.

A total of 37 patients were evaluated with at least 1 target lesion that was observed in imaging at 24 weeks before receiving the first dose of treatment. Results were assessed by independent radiological review and efficacy was defined as a 20% or more reduction in target lesion volume from the baseline in up to 3 lesions; this needed to be confirmed by at lease 1 subsequent imaging assessment.

The recommended dose for patients aged 2 to 18 was 50 mg of alpelisib orally once daily with food. For pediatric patients 6 years and older, the dose can be increased to 125 mg after 24 weeks. The dosing for adult patients 18 years and older is 250 mg orally taken once daily with food.

The primary end point of the study was the proportion of patients with a response at 24 weeks, and the secondary end point was percent of change in the measure of target lesion. Patients were eligible for treatment if they had a documented diagnosis of PROS, evidence of PI3K-mutated gene, and a severe or life-threatening condition where treatment was necessary.

At week 24, 27% (95% CI, 14%-44%) of patients had a radiological response, and 60% had a response the lasted for 10 months or longer. The most common adverse effects observed in 10% or more of patients included diarrhea, stomatitis, and hyperglycemia.

Alpelisib has previously been granted priority review, breakthrough designation, and orphan drug designation for patients with of PIK3CA-realted overgrowth spectrum who require systemic therapy.

References

  1. FDA approves alpelisib for PIK3CA-related overgrowth spectrum. News Release. FDA. April 5, 2022. Accessed April 6, 2022. https://bit.ly/35M2Swv
  2. National Institute of Health. PIK3CA-related overgrowth spectrum. Revised July 10, 2018. Accessed April 6, 2022. https://bit.ly/3Kk3tEs
Recent Videos
Co-hosts Kristie L. Kahl and Andrew Svonavec highlight what to look forward to at the 2025 ASCO Annual Meeting, from hot topics and emerging trends to travel recommendations.
The pediatric care wing of the Jack & Sheryl Morris Cancer Center is intended to accommodate the physical and emotional needs of younger patients with cancer.
Having all the necessary staff together, from medical oncologists to pharmacists, helps deliver the best possible outcomes to patients with cancer.
Joseph C. Landolfi, DO, CPE, and Michelle Morrison, MPH, BSHA, RN, discuss how they can use their leadership roles to improve cancer care.
Leadership of a new cancer center as part of JFK University Medical Center discuss how they can support frontline clinicians.
Related Content
Advertisement

The addition of olanzapine to antiemetics might alleviate insomnia, appetite loss, anxiety, and depression in those receiving concurrent chemoradiation.

Olanzapine May Prevent Radiation-Induced Nausea and Vomiting

Roman Fabbricatore
June 3rd 2025
Article

The addition of olanzapine to antiemetics might alleviate insomnia, appetite loss, anxiety, and depression in those receiving concurrent chemoradiation.


Professor of Pharmacology, Eric Winer, MD, spoke about a publication he authored exploring the state of oncologist burnout and how it impacts practice.

Exploring Burnout Causes and Management in Oncologic Practice

Eric P. Winer, MD
June 2nd 2025
Podcast

Professor of Pharmacology, Eric Winer, MD, spoke about a publication he authored exploring the state of oncologist burnout and how it impacts practice.


Experts highlight research at the 2025 ASCO Annual Meeting that may move the needle in gynecologic cancers, hematologic malignancies, and other fields.

ASCO 2025: Key Anticipated Updates Across Cancer Care

Russ Conroy
May 28th 2025
Article

Experts highlight research at the 2025 ASCO Annual Meeting that may move the needle in gynecologic cancers, hematologic malignancies, and other fields.


Experts in gynecologic cancer, genitourinary malignancies, and other disciplines highlight noteworthy clinical data slated for presentation at ASCO 2025.

Spotlighting Key Upcoming Presentations Across Oncology at ASCO 2025

Rachel N. Grisham, MD;MinhTri Nguyen, MD;Eric Singhi, MD;Douglas Adkins, MD;Benjamin Garmezy, MD
May 26th 2025
Podcast

Experts in gynecologic cancer, genitourinary malignancies, and other disciplines highlight noteworthy clinical data slated for presentation at ASCO 2025.


CHMP Recommends Mirdametinib for EU Approval in Adult/Pediatric NF1-PN

CHMP Recommends Mirdametinib for EU Approval in Adult/Pediatric NF1-PN

Tim Cortese
May 23rd 2025
Article

The EMA’s CHMP has adopted a positive opinion for mirdametinib in patients with inoperable plexiform neurofibromas of neurofibromatosis type 1.


GLP-1 Use Not Linked to Increased Obesity-Related Cancer Rates

GLP-1 Use Not Linked to Increased Obesity-Related Cancer Rates

Ariana Pelosci
May 22nd 2025
Article

There was a 7% lower incidence of obesity-related cancer with GLP-1 receptor agonist use.

Related Content
Advertisement

The addition of olanzapine to antiemetics might alleviate insomnia, appetite loss, anxiety, and depression in those receiving concurrent chemoradiation.

Olanzapine May Prevent Radiation-Induced Nausea and Vomiting

Roman Fabbricatore
June 3rd 2025
Article

The addition of olanzapine to antiemetics might alleviate insomnia, appetite loss, anxiety, and depression in those receiving concurrent chemoradiation.


Professor of Pharmacology, Eric Winer, MD, spoke about a publication he authored exploring the state of oncologist burnout and how it impacts practice.

Exploring Burnout Causes and Management in Oncologic Practice

Eric P. Winer, MD
June 2nd 2025
Podcast

Professor of Pharmacology, Eric Winer, MD, spoke about a publication he authored exploring the state of oncologist burnout and how it impacts practice.


Experts highlight research at the 2025 ASCO Annual Meeting that may move the needle in gynecologic cancers, hematologic malignancies, and other fields.

ASCO 2025: Key Anticipated Updates Across Cancer Care

Russ Conroy
May 28th 2025
Article

Experts highlight research at the 2025 ASCO Annual Meeting that may move the needle in gynecologic cancers, hematologic malignancies, and other fields.


Experts in gynecologic cancer, genitourinary malignancies, and other disciplines highlight noteworthy clinical data slated for presentation at ASCO 2025.

Spotlighting Key Upcoming Presentations Across Oncology at ASCO 2025

Rachel N. Grisham, MD;MinhTri Nguyen, MD;Eric Singhi, MD;Douglas Adkins, MD;Benjamin Garmezy, MD
May 26th 2025
Podcast

Experts in gynecologic cancer, genitourinary malignancies, and other disciplines highlight noteworthy clinical data slated for presentation at ASCO 2025.


CHMP Recommends Mirdametinib for EU Approval in Adult/Pediatric NF1-PN

CHMP Recommends Mirdametinib for EU Approval in Adult/Pediatric NF1-PN

Tim Cortese
May 23rd 2025
Article

The EMA’s CHMP has adopted a positive opinion for mirdametinib in patients with inoperable plexiform neurofibromas of neurofibromatosis type 1.


GLP-1 Use Not Linked to Increased Obesity-Related Cancer Rates

GLP-1 Use Not Linked to Increased Obesity-Related Cancer Rates

Ariana Pelosci
May 22nd 2025
Article

There was a 7% lower incidence of obesity-related cancer with GLP-1 receptor agonist use.

Advertisement
About
Advertise
CureToday.com
OncLive.com
OncNursingNews.com
TargetedOnc.com
Editorial
Contact
Terms and Conditions
Privacy
Do Not Sell My Personal Information
Contact Info

2 Commerce Drive
Cranbury, NJ 08512

609-716-7777

© 2025 MJH Life Sciences

All rights reserved.