The FDA’s 2024 PDUFA calendar is far from complete, but already it contains several dates worth watching, from the first new drug class for schizophrenia in decades, to a second indication for a history-making CRISPR treatment.
This follows a year in which the FDA approved 55 new drugs, including several firsts, like the first vaccine against RSV and the first oral medication for postpartum depression.
Here are four PDUFA dates to watch in 2024.
A tumor infiltrating therapy for advanced melanoma
Developer: Iovance Biotherapeutics
Drug: lifileucel
PDUFA date: Feb. 24
Why it matters: Although its first PDUFA date was delayed by resource constraints at the FDA, Iovance Biotherapeutics’ advanced melanoma treatment has the potential to be the first and only tumor infiltrating lymphocyte (TIL) cell therapy for patients with advanced melanoma, as well as the first one-time cell therapy for a solid tumor cancer. The FDA already granted the drug priority review and a regenerative medicine advanced therapy designation. However, Iovance shares tumbled late last month after a patient died during a clinical trial for another of its investigational TIL therapies, LN-145, and the FDA placed a clinical hold on the trial. The company said the clinical hold had no impact on lifileucel or its PDUFA date.
A second indication for the history-making CRISPR treatment
Developer: Vertex Pharmaceuticals and CRISPR Therapeutics
Drug: Casgevy
PDUFA date: March 30
Why it matters: The buzzy gene editing drug dubbed Casgevy already made history when a year-end approval for sickle cell disease made it the first-ever FDA approved CRISPR-based treatment. Now, Vertex and CRISPR Therapeutics are going after an approval for the same drug to treat another inherited blood disorder: transfusion-dependent beta-thalassemia (TDT). Beta-thalassemia patients don’t make enough beta globin, and the transfusion-dependent form of the disease is the most severe, which creates a huge burden for regular blood transfusions.
In the wider gene editing field, Casgevy also remains the only CRISPR frontrunner, with 88% of other CRISPR drugs in early development and unlikely to be submitted for an approval in the near future, according to GlobalData.
A potential first-in-class drug for a difficult cancer
Developer: Amgen
Drug: tarlatamab
PDUFA date: June 12
Why it matters: The FDA gave priority review to Amgen’s advanced small cell lung cancer drug, which uses a patient's own T cells to fight tumor-specific antigens. The drug, first in its class, is meant for adults who have already tried two or more prior lines of treatment. Small cell lung cancer is aggressive, relatively common (affecting 15% of the more than 2.2 million patients diagnosed with lung cancer worldwide each year, Amgen said) and incredibly prone to post-treatment relapse, making the potential market for tarlatamab a big one. The FDA granted tarlatamab breakthrough therapy designation.
The first treatment for schizophrenia in decades
Developer: Karuna Therapeutics
Drug: KarXT
PDUFA date: Sept. 26
Why it matters: If approved, KarXT would end a decades-long wait for a new schizophrenia treatment. The drug not only improved schizophrenia symptoms in clinical trials but “was not associated with” the common and bothersome side effects in currently available antipsychotics, including weight gain, sleepiness and movement disorders, Karuna said.
The drug’s market potential also caught the attention of Bristol Myers Squibb, which recently announced it will acquire Karuna Therapeutics in a $14 billion deal.
Unlike existing treatments, KarXT has a novel mechanism of action that doesn’t directly block dopamine receptors. Instead, it’s a dual M1/M4 muscarinic acetylcholine receptor agonist in the central nervous system.
