In the first part of a two-part interview, Paz Vellanki of Precision for Medicine explains what sponsors should bring to early FDA meetings and how manufacturing and assay readiness can delay rare disease trials.
|Articles|March 6, 2008
FDA Approves Regeneron's Acralyst
Author(s)Chitra Sethi
The US FDA has approved Arcalyst (rilonacept, an interleukin-1 blocker) for the long-term treatment of two cryopyrin-associated periodic syndrome (CAPS) disorders.
Advertisement
The US FDA has approved Arcalyst (rilonacept, an interleukin-1 blocker) for the long-term treatment of two cryopyrin-associated periodic syndrome (CAPS) disorders: familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS). Arcalyst, manufactured by Regeneron Pharmaceuticals, Inc. (Tarrytown, NY), is the first treatment for rare inflammatory syndromes.
Arcalyst blocks interleukin-1, which is a signaling protein secreted by certain immune-related cells in the body. Interleukin-1 acts as a messenger to regulate inflammatory responses, but in excess it can be harmful and has been shown to be key in the inflammation seen in CAPS sufferers with FCAS or MWS.
The FDA issued its approval following a priority review, which speeds the review process for patients who have unmet medical needs. Regeneron expects to launch Arcalyst within the next 30 days.
Advertisement
Related to this article
FDA clears the IND for Biolojic Design’s BD200, an AI-designed Trop2/Nectin-4 multibody ADC; Blackstone Life Sciences agrees to provide Pfizer up to $1.5 billion to develop its monthly GLP-1 receptor agonist berobenatide; and enGene Therapeutics plans a year-end BLA for detalimogene voraplasmid in BCG-unresponsive non-muscle invasive bladder cancer.
Detalimogene voraplasmid produced a 56% complete response rate in BCG-unresponsive bladder cancer, with an estimated 43% of responses lasting a year.
FDA has cleared Biolojic Design’s IND for BD200, a bispecific antibody-drug conjugate targeting Trop2 and Nectin-4 whose arms can each bind either target. A global Phase 1 study in solid tumors is expected to begin in the coming weeks.
Blackstone Life Sciences will fund up to $1.5 billion of development costs through 2028 for Pfizer’s monthly GLP-1 receptor agonist berobenatide, in exchange for milestone payments tied to approval and sales. Pfizer keeps full control of the program.
Gene-corrected nula-cel freed all 3 RESTORE patients from transfusions, with sickle hemoglobin below 20% in the 2 followed for over 6 months.
Beam Therapeutics’ base-edited cell therapy risto-cel meets its primary endpoint in the BEACON sickle cell disease trial, Amgen’s CD40L antagonist dazodalibep reduces systemic disease activity in the phase 3 OASIZ-301 Sjögren’s disease trial, and ImvivaBio’s allogeneic CD19/BCMA CAR-T CTA313 sustains lupus remission through 12 months.
Matt Truppo, Richard Jaenisch, and Brian Drapeau examine AI's impact on drug success, human oversight, data governance, and biopharma's future.
Advertisement
Advertisement
Trending on BioPharm International
1
Blackstone to Provide Pfizer Up to $1.5 Billion for Monthly GLP-1 Berobenatide Development
2
ImvivaBio’s Dual-Target Allogeneic CAR-T Sustains Lupus Remission Through 12 Months
3
Updates on Simtra BioPharma Solutions’ Bloomington Capacity Expansions
4
The BioPharm Brief: An AI-Designed Multibody ADC Enters the Clinic, Blackstone Backs Pfizer’s Monthly GLP-1, and enGene Readies a Bladder Cancer BLA
5