FDA Drug Approval Decisions Expected in May 2024

The Prescription Drug User Fee Act (PDUFA) date refers to the deadline set by the US Food and Drug Administration (FDA) for reviewing a New Drug Application (NDA) or Biologics License Application (BLA) and making a final decision on marketing approval. The typical period for review is 10 months after the drug application has been accepted by the Agency. For drugs that have Priority Review, the review period is reduced to 6 months from the time of application acceptance.

TransCon PTH for Hypoparathyroidism

PDUFA date: May 14, 2024

TransCon PTH (palopegteriparatide) is an investigational, once-daily, long-acting prodrug of parathyroid hormone (PTH[1-34]) designed to restore physiologic levels of PTH for 24 hours. The NDA is supported by data from the phase 3 PaTHway trial (ClinicalTrials.gov Identifier: NCT04701203) and the phase 2 PaTH Forward trial (ClinicalTrials.gov Identifier: NCT04009291), which evaluated the efficacy and safety of TransCon PTH in adults with hypoparathyroidism. Findings from the PaTHway trial showed a statistically significantly greater proportion of patients treated with TransCon PTH met the primary endpoint achieving serum calcium levels within normal range and independence from therapeutic levels of conventional therapy compared with placebo. In the PaTH Forward trial, long-term treatment with TransCon PTH provided a durable response, with 93% of patients achieving independence from conventional therapy with active vitamin D and therapeutic levels of calcium through week 110.

Rivoceranib Plus Camrelizumab for Unresectable Hepatocellular Carcinoma

PDUFA date: May 16, 2024

Rivoceranib is an orally-administered inhibitor of vascular endothelial growth factor receptor 2. Camrelizumab is a humanized monoclonal antibody that targets programmed death-1 (PD-1) receptor and is administered intravenously. The NDA is supported by data from the phase 3 CARES 310 study (ClinicalTrials.gov Identifier: NCT03764293), which compared the efficacy and safety of rivoceranib plus camrelizumab to sorafenib in 543 adults with advanced hepatocellular carcinoma who had not previously received systemic therapy. Findings showed rivoceranib plus camrelizumab demonstrated statistically significant and clinically meaningful prolonged overall survival and progression free survival vs sorafenib.

Lisocabtagene Maraleucel for Follicular Lymphoma

PDUFA date: May 23, 2024

Lisocabtagene maraleucel (liso-cel) is a CD19-directed genetically modified autologous T cell immunotherapy. It is currently approved under the brand name Breyanzi^® for adult patients with large B-cell lymphoma. The sBLA is supported by data from the TRANSCEND FL trial (ClinicalTrials.gov Identifier: NCT04245839), an open-label, phase 2 single-arm study evaluating liso-cel in patients with relapsed or refractory indolent B cell non-Hodgkin lymphoma, including high-risk second-line FL. Among the 101 relapsed/refractory FL patients in TRANSCEND FL, the overall response rate was 97% (95% CI, 91.6-99.4; P <.0001), with 94% achieving a complete response. At a median follow-up of 16.6 months, median duration of response was not reached; 81.9% of responders had an ongoing response at 12 months. At a median follow-up of 17.5 months, median progression free survival (PFS) was also not reached; 12 month PFS was achieved in 80.7% of patients.

Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa

PDUFA date: May 25, 2024

Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by extensive blistering and severe skin wounds. It is caused by a mutation in the COL7A1 gene, resulting in the inability to produce type VII collagen. Prademagene zamikeracel (pz-cel) is an autologous cell therapy that consists of epidermal sheets that deliver functional COL7A1 genes into the patient’s own skin cells to enable normal type VII collagen expression and facilitate wound healing. The application is supported by data from the phase 3 VIITAL study (ClinicalTrials.gov Identifier: NCT04227106), which included 43 large chronic wound pairs in 11 patients with RDEB. Findings showed 81.4% of pz-cel-treated wounds achieved 50% or greater wound healing compared with 16.3% of untreated control wounds (P <.001). Additionally, a statistically significant improvement in pain reduction associated with wound dressing changes was observed with pz-cel when compared with untreated control wounds (P =.0002).

Lisocabtagene Maraleucel for Mantle Cell Lymphoma

PDUFA date: May 31, 2024

In addition to the follicular lymphoma indication, the FDA is expected to make a decision on the use of lisocabtagene maraleucel (liso-cel) for mantle cell lymphoma (MCL). The sBLA includes data from the TRANSCEND NHL-001 trial (ClinicalTrials.gov Identifier: NCT02631044), an open-label, phase 1, single-arm study assessing liso-cel in in a cohort of patients with relapsed or refractory MCL. The MCL cohort of TRANSCEND NHL-001 enrolled patients with relapsed or refractory disease after 2 or more prior lines of therapy, including a Bruton tyrosine kinase inhibitor (n=74). At a median follow-up of 16.1 months, the objective response rate was 86.5% (95% CI, 76.5-93.3; P <.0001), with 74.3% of patients achieving a complete response.