The Food and Drug Administration (FDA) is expected to make several landmark decisions this quarter. While they may not have direct implications for Australia or any Therapeutic Goods Administration (TGA) decisions, they are certainly worth keeping an eye on.
The TGA is known to consider and monitor the use of real-world data and real-world evidence to support regulatory decision-making for drug and biological products.
So what should we be on the lookout for over the next few months?
Two significant biotechnology acquisitions last year centred on innovative treatments for schizophrenia, with the first of these therapies potentially gaining FDA approval by the end of September. This milestone could underscore the growing interest in psychiatric medications.
Additional novel psychiatric treatments are progressing through trials, including psychedelics such as MDMA and psilocybin. The FDA faces a challenging decision, as its advisory panel recently criticised the use of MDMA-assisted psychotherapy for post-traumatic stress disorder (PTSD).
Meanwhile, Gilead Sciences might secure approval for a drug acquired through its CymaBay Therapeutics deal, and a new cell therapy could enter the market.
MDMA-based therapy for PTSD
Lykos Therapeutics is aiming to build a business around MDMA, commonly known as ecstasy, for PTSD treatment.
Despite promising trial results, FDA advisers expressed concerns about safety and trial conduct, casting doubt on approval prospects. The FDA is expected to decide by August 11.
KarXT for schizophrenia
A groundbreaking antipsychotic, KarXT, developed by Karuna Therapeutics, could become the first new form of treatment for schizophrenia in decades.
Clinical trials demonstrated significant symptom reduction and a rapid onset of effects.
While KarXT has side effects, it avoids the excessive weight gain and movement issues associated with older antipsychotics.
Analysts anticipate FDA approval by September 26, with potential peak annual sales exceeding US$10 billion.
TransCon PTH for hypoparathyroidism
Ascendis Pharma’s TransCon PTH, a replacement therapy for hypoparathyroidism, could be approved by August 14.
Despite previous setbacks due to manufacturing concerns, recent positive study results and approvals in Europe and the UK bolster its prospects.
afami-cel for synovial sarcoma
Adaptimmune’s afami-cel, a TCR cell therapy for the rare cancer synovial sarcoma, could gain FDA approval by August 4.
While the company has faced financial challenges, a successful approval may validate its approach, though strong commercial performance will be crucial for sustained success.
seladelpar for primary biliary cholangitis
Gilead’s acquisition of CymaBay brought the experimental liver disease treatment, seladelpar, into its portfolio.
Expected to address the chronic condition primary biliary cholangitis, seladelpar’s approval decision is due by August 14.
Gilead highlights its effectiveness in reducing associated itching and anticipates moderate sales contributions.