The rare disease space is awaiting two FDA verdicts in February, one for a genetic disease and another for a non-malignant tumor.
The FDA is looking at five big deadlines in February, including one for a broad meningococcal vaccine that promises to provide potent protection against five of the most common serogroups in the U.S.
Read below for more.
Indivior Proposes Alternate Injection Sites for Opioid Use Disorder Therapy
By February 7, the FDA is set to release its decision on Indivior’s Prior Approval Supplement (PAS) filing, which proposes alternative injection sites for its opioid use disorder treatment Sublocade (buprenorphine extended-release).
Sublocade is a long-acting formulation of buprenorphine, an opioid agonist, that is indicated for patients with moderate to severe opioid use disorder who have already initiated treatment with another buprenorphine-containing product. Currently, the drug is administered through a subcutaneous injection in the abdomen.
If approved, the PAS will allow sublocade to be delivered through the thigh, buttocks and back of the upper arm. The PAS also proposes a rapid induction protocol that would shorten induction time from the presently approved minimum of 7 days, down to a single dose of transmucosal buprenorphine followed by a 1-hour observation period.
Indivior is backing its PAS with data showing the safety and efficacy of the proposed routes of administration and rapid induction protocol.
“These updates are expected to enhance the patient experience and expand treatment access especially for those patients who … have long-term opioid use including fentanyl,” the biotech wrote in its October 2024 news release.
GSK Looks to Expand Meningococcal Protection with New Shot
GSK is advancing a 5-in-1 vaccine candidate designed to elicit broad protection against the five most common bacterial groups causing invasive meningococcal disease. The FDA is currently reviewing the pharma’s Biologics License Application (BLA), with a decision due on Feb. 14.
The investigational shot, dubbed MenABCWY, combines two well-established meningococcal vaccines: Bexsero, which targets group B bacteria, and Menveo, which covers the A, C, W and Y serogroups. Both Bexsero and Menveo are owned by GSK.
According to the pharma, combining these five bacterial groups in one protective shot “aims to reduce the number of injections, simplifying immunization” and improving adherence and completion rates for the vaccine. In turn, this can lead to better health outcomes and lower the overall incidence of invasive meningococcal disease, GSK said in a statement.
To back MenABCWY, GSK provided Phase III data showing that the vaccine candidate induced non-inferior immune responses as compared with one dose of Menveo and with two doses of Bexsero. MenABCWY was also well-tolerated and its adverse events were consistent with the two comparator vaccines.
Ono Sets Sights on Rare, Non-Malignant Tumor
Osaka-based Ono Pharmaceutical is proposing vimseltinib, an investigational colony stimulating factor 1 receptor (CSF1R) inhibitor, for the treatment of tenosynovial giant cell tumor (TGCT). The FDA is expected to release its decision by Feb. 17.
TGCT is a rare type of non-malignant tumor that develops in or around joints. The disease is caused by a mutation in the CSF1 gene that results in the overexpression of the protein, in turn recruiting an abundance of CSF1R-expressing inflammatory cells to the tumor site. TGCT is benign but can damage surrounding tissue once progressed. Patients often suffer from pain, swelling and limitations in joint movement.
Currently, TGCT is managed primarily through surgery, though this option frequently leads to disease recurrence. In patients who cannot undergo surgery, systemic therapies are limited, according to Ono. If left unchecked or allowed to continuously recur, TGCT can lead to significant disability.
Vimseltinib is an orally available switch-control tyrosine kinase inhibitor designed to strongly block CSF1R, targeting a key disease pathway in TGCT. Ono, through its subsidiary Deciphera, is backing vimseltinib’s application with data from the pivotal Phase III MOTION trial, which found that the candidate elicits significantly better objective response rate versus placebo, while also hitting its key secondary endpoints.
SpringWorks Awaits Verdict for Rare Genetic Disease Therapy
By Feb. 28, the FDA is expected to decide on SpringWorks Therapeutics’ investigational MEK inhibitor mirdametinib, which the Connecticut-based biotech is developing for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) in adults and children.
NF1 is a highly heterogeneous rare and genetic disease with varied manifestations across different organ systems, including tumor formation and growth, neurological complications and skeletal deformities. The PN subtype of NF1 is characterized by tumors along the sheathes of peripheral nerves, in turn leading to severe disfigurement, functional impairment and pain, according to SpringWorks.
NF1, including the PN subtype, is caused by loss-of-function mutations in the NF1 gene, which under healthy conditions encodes for the neurofibromin protein that suppresses the MAPK pathway. Mirdametinib addresses this by penetrating into the central nervous system to inhibit MEK1 and MEK2, two signaling molecules that play key roles in the MAPK pathway.
To back its drug application, SpringWorks provided data from the Phase IIb ReNeu study, which focused on patients aged 2 years and older and found that mirdametinib elicited “robust” objective response rates, deep and durable responses and notable improvements in pain and health-related quality of life. The FDA has granted mirdametinib its Priority Review and Orphan Drug designations for NF1-PN.
Eton Pharma’s New Oral Hydrocortisone Formulation Nears Approval
Also by Feb. 28, the FDA is scheduled to release its verdict regarding Eton Pharmaceuticals’ novel oral formulation of hydrocortisone, dubbed ET-400, for adrenal insufficiency in infants.
ET-400 is a new and proprietary formulation of oral hydrocortisone that is stable at room temperature. Eton recently won patent protection over this formulation that will last until 2043. The biotech also has other patent applications for ET-400 that are currently under review.
Eton also owns Alkindi Sprinkle, an oral granule formulation of hydrocortisone that is likewise indicated for children with adrenocortical insufficiency. The drug, approved in 2020, is an immediate-release glucocorticoid that can sufficiently replace cortisol in these patients. Alkindi Sprinkle is specifically formulated to allow parents to give lower doses to small children, instead of needing to cut higher-strength hydrocortisone tablets.
If approved, ET-400 would allow Eton to “capture a greater percentage of the oral hydrocortisone market,” CEO Sean Brynjelsen said in the biotech’s press statement on the FDA’s acceptance of ET-400’s drug application. Together with Alkindi Sprinkle, ET-400 could hit peak sales of more than $50 million per year, Brynjelsen added.
