- UPDATED: Wednesday, May 20 at 11:20 a.m.
- ETPatrick Soon-Shiong’s ImmunityBio has convinced the FDA to weigh its case for a potential expansion of its bladder cancer med Anktiva, citing “overlapping features” in the indication it’s targeting with the interleukin-15 agonist’s existing approval. The FDA has now accepted ImmunityBio’s application for Anktiva plus the Bacillus Calmette-Guérin (BCG) vaccine in patients with BCG-unresponsive non-muscle-invasive bladder cancer (NMIBC) with papillary disease without carcinoma in situ (CIS).
- The FDA has set a target decision date of Jan.
UPDATED: Wednesday, May 20 at 11:20 a.m. ET
Patrick Soon-Shiong’s ImmunityBio has convinced the FDA to weigh its case for a potential expansion of its bladder cancer med Anktiva, citing “overlapping features” in the indication it’s targeting with the interleukin-15 agonist’s existing approval.
The FDA has now accepted ImmunityBio’s application for Anktiva plus the Bacillus Calmette-Guérin (BCG) vaccine in patients with BCG-unresponsive non-muscle-invasive bladder cancer (NMIBC) with papillary disease without carcinoma in situ (CIS). The FDA has set a target decision date of Jan. 6, 2027, ImmunityBio said in a May 19 release.
The company is buoying its application with data from its phase 2/3 QUILT 3.032 trial, which met its primary endpoint with Anktiva achieving a 12-month disease-free survival rate of 58.2%, per ImmunityBio.
The acceptance follows a "Refusal to File" letter that the FDA sent to ImmunityBio a year ago for the same indication.
The problem centered on whether ImmunityBio's single-arm study design measuring a time-to-event endpoint is sufficient for a filing. At the time, ImmunityBio said it was shocked, accusing the FDA of an about-face on a previous agreement. However, citing people with knowledge of the FDA's side of this story, Stat reported that the agency had never endorsed the company's plan back then.
ImmunityBio won its first approval in Anktiva for BCG-unresponsive NMIBC with CIS in early 2024.
The company’s expansion bid hinges on the argument that CIS and papillary NMIBC share biological similarities, which was the topic of an FDA public workshop earlier this week, the company pointed out.
“At the workshop,” ImmunityBio said in its release, “panelists stated that CIS and papillary disease arise from the same cancer inducing clone, is therefore the same disease and the clinical decision treatment made when papillary disease alone is identified in the real-world is to treat the patient with already FDA approved therapies for CIS and papillary disease.”
UPDATED: Monday, May 18 at 2:30 p.m. ET
The approval was based on results from an investigator-initiated phase 2 study that assessed the efficacy and safety of Alecensa in adult and pediatric patients with rare cancers with advanced or recurrent ALK gene abnormalities.
Japan also has given a thumbs up to Boehringer Ingelheim’s Jascayd as a treatment for adults with either idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF).
The FDA approved Jascayd with separate nods for the lung conditions two months apart in the fourth quarter of last year.
Jascayd is the first phosphodiesterase 4B (PDE4B) inhibitor with antifibrotic and immunomodulatory effects approved in these indications. Pooled analysis of trials in both indications showed a 59% reduction in the risk of death provided by Jascayd versus placebo.
Also in Japan, its Ministry of Health, Labour and Welfare (MHLW) has signed off on an expanded approval for GSK’s respiratory syncytial virus (RSV) vaccine Arexvy for those ages 18 to 49 who are at an increased risk of developing severe disease because of chronic conditions.
Arexvy has been approved in Japan for those age 60 and older and for those ages 50 to 59 who are at increased risk. Arexvy was originally approved in the U.S., Europe and Japan in 2023.
Along with the expansion, prescribing information has been updated to include immuno-compromised patients among those at increased risk, helping clinicians identify who may benefit from RSV prevention.
UPDATED: Friday, May 15 at 10:25 a.m. ET
England's National Institute for Health and Care Excellence (NICE) is backing NHS coverage of AstraZeneca's Imfinzi as part of a perioperative treatment for stomach cancer that has not spread extensively and can be removed through surgery.
The decision was made just 17 days after an approval from the country's Medicines and Healthcare products Regulatory Agency (MHRA) under a simpler assessment process, which does not require a committee meeting, according to NICE.
The coverage is based on results from the phase 3 Matterhorn trial in stage 2 to 4a resectable gastric cancer and gastroesophageal junction (GEJ) cancer. The addition of Imfinzi both before and after surgery to chemo led to a 29% improvement in event-free survival compared to presurgical chemotherapy alone.
More than 1,500 people in England will be able to benefit from the regimen per year, NICE said.
Meanwhile, in a final guidance, NICE has endorsed Merck & Co.'s Winrevair, used with other pulmonary arterial hypertension (PAH) treatments, for adults at World Health Organization functional class (WHO FC) 2 or 3 stages of disease. These patients have low, intermediate-low or intermediate-high-risk PAH.
Clinical data show that Winrevair plus background treatments improves exercise capacity and WHO FC status compared with placebo plus background treatments, NICE noted (PDF).
The list price of Winrevair is 5,422.50 pound sterling for a 45-mg vial and 7,230 pound sterling for a 60-mg vial. Merck has agreed to a confidential discount to secure its coverage.
NICE this week also approved routine access to Biogen's Spinraza and Roche's Evrysdi for certain people with spinal muscular atrophy (SMA).
"The decision marks a significant milestone after both medicines were previously made available through managed access agreements while additional evidence on their effectiveness was gathered," NICE noted in a May 14 release.
Both clinical data and real-world experiences of patients were considered. Patients reported improved physical and mental wellbeing, fewer emergency hospital admissions—particularly for respiratory infections—and greater participation in education, employment and social activities, NICE noted. Their caregivers reported better mental health, improved sleep and an increased ability to balance caring responsibilities with work and family life.
Spinraza is covered to treat both pre-symptomatic and symptomatic SMA in patients who have not responded to Novartis' gene therapy Zolgensma. Evrysdi is now offered to Zolgensma non-responders with SMA types 1, 2 or 3, as well as for pre-symptomatic cases where the gene therapy is unsuitable.
