The FDA has four big events in the coming two weeks, including an advisory panel meeting for an ultra-rare disease.
The FDA will welcome August with four key events over the next two weeks: three target action dates for experimental treatments and one advisory committee meeting for an ultra-rare neurodegenerative disease.
Read below for more.
Adaptimmune Eyes First Approval of Engineered T Cell Therapy in Solid Tumors
By August 4, the FDA is set to release its verdict on Adaptimmune Therapeutics’ Biological License Application (BLA) for afami-cel, an investigational engineered T cell therapy the biotech is proposing for the treatment of advanced synovial sarcoma.
The regulator has granted afami-cel Priority Review, which cuts down the review process from the standard 10 months to six months.
Afami-cel works by targeting and binding to melanoma-associated antigen A4 (MAGE-A4), a protein that plays a central role in preventing cell cycle arrest and blocking cell death. MAGE-A4 is highly expressed in solid tumors, especially synovial sarcoma.
Adaptimmune’s BLA, for which it completed the rolling submission in December 2023, is backed by data from the first cohort of the pivotal SPEARHEAD-1 trial. In March 2024, the biotech published study results in The Lancet demonstrating an overall response rate of 39% in synovial sarcoma patients.
As for safety, SPEARHEAD-1 documented 37 cases of cytokine release syndrome, corresponding to an occurrence rate of 71%, though only one episode was grade 3 in severity. There were no treatment-related deaths.
If approved, afami-cel could be the “first engineered T-cell therapy for solid tumors and the first effective treatment option for synovial sarcoma in more than a decade,” according to Adaptimmune’s press release announcing the FDA’s acceptance of its BLA.
scPharma Seeks to Expand Furoscix Injection
scPharmaceuticals is proposing an expanded label for its subcutaneous injection Furoscix (furosemide) to allow its use in heart failure patients with New York Heart Association (NYHA) Class IV disease. The FDA’s decision is due on August 10.
Administered through a single-use on-body infuser, Furoscix delivers an initial 30-mg dose of furosemide over the first hour, followed by 12.5 mg of the drug per hour for the next four hours, according to its label. Furoscix works by blocking the reabsorption of sodium in a specific area of the kidney, which in turn promotes the production of urine. In patients with heart failure, this mechanism of action helps prevent fluid retention and overload. Furoscix also helps with vasodilation.
Currently, Furoscix is indicated only for chronic heart failure patients of NYHA Classes II and III. In August 2023, however, scPharma completed a Type C meeting with the FDA, during which the company received “positive” feedback from the regulator regarding the potential expansion of Furoscix’s label to also cover NYHA Class IV patients.
At the time, CEO John Tucker said the positive Type C meeting “paves the way for potential expansion of the Furoscix indication” into NYHA Class IV patients. “We estimate that as many as 10% of all heart failure patients are Class IV, and a meaningful percentage of these—as many as 40%—may benefit from Furoscix,” he said.
Lykos Awaits FDA Verdict on MDMA Therapy for PTSD
Lykos Therapeutics is advancing an investigational MDMA-assisted therapy for post-traumatic stress disorder (PTSD), which uses oral MDMA tablets in conjunction with supportive mental health services. The FDA’s decision is due on August 11.
Lykos is backing its application with data from the Phase III MAPP1 and MAPP2 studies, both of which met their primary efficacy endpoints. The MDMA-based regimen led to significant improvements in the Clinician-Administered PTSD Scale for DSM-5, a validated tool used to diagnose and evaluate the symptoms of PTSD.
The trials also hit their secondary endpoints, with MDMA treatment easing functional impairments associated with PTSD. The investigational regimen was also safe overall and was not linked with serious adverse events.
Despite these data, however, Lykos’ MDMA-assisted therapy has recently been the subject of controversy. In May 2024, the Institute for Clinical and Economic Review (ICER) flagged substantial uncertainties in the company’s clinical development programs, noting that its studies were “essentially, unblinded” because of MDMA’s psychedelic effects.
ICER also revealed it had received reports that some study participants had been pressured to keep the results of Lykos’ studies “favorable.”
A couple of weeks later, the FDA’s Psychopharmacologic Drugs Advisory Committee cited these issues in voting overwhelmingly against Lykos. Split 9–2, the panel of external experts found that the company had not provided enough data to back its MDMA-based candidate. The panel voted 10–1 against Lykos on the question of risk/benefit profile.
Zevra’s NPC Drug to Face Newly Formed FDA Adcomm
On August 2, the FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) will convene to discuss the New Drug Application for Zevra Therapeutics’ oral drug candidate arimoclomol for the treatment of Niemann-Pick disease type C (NPC).
NPC is an ultra-rare, progressive and neurodegenerative lysosomal storage disease characterized by severe physical and cognitive impairmentsalongside other neurological deficits manifesting as speech, motor and swallowing problems. NPC is irreversible and can be fatal within months.
NPC is caused by mutations in the NPC1 or NPC2 genes, which in turn compromise the body’s ability to transport and clear cholesterol and other lipids within the cell. As a result, these molecules accumulate pathologically in various tissues and organs across the body, including the brain. There are currently no approved treatments for NPC.
Designed to cross the blood-brain barrier, arimoclomol helps make cells more robust and prevents their destruction even when under stress. The drug candidate was previously rejected by the FDA in June 2021, when it was still owned by Orphazyme.
In its resubmission, which it filed in December 2023, Zevra addressed the regulator’s prior concerns by providing additional data to support arimoclomol’s mechanism of action. The biotech also performed non-clinical, natural history and real-world studies to better establish the efficacy and safety of arimoclomol in NPC.
