Regulatory

The importance of pre-approval information exchange (PIE) with payers and other strategic considerations to help navigate today’s market access challenges and regulatory requirements in bringing promising cell and gene therapies to the market.

UGN-102 has the potential to be the first FDA-approved treatment for low-grade intermediate-risk non-muscle invasive bladder cancer, UroGen says.

Approval of Nemluvio was based on positive results from the Phase III OLYMIA trials, which demonstrated significant itch reductions.

The Complete Response Letter stated that current data on midomafetamine capsules is insufficient for approval in treating patients with post-traumatic stress disorder.

Yorvipath is designed to parathyroid hormone exposure over 24 hours in patients with hypoparathyroidism.

Approval of neffy marks a significant advancement in epinephrine delivery, offering a less painful alternative to traditional needle injections.

Lymphir is the first therapy to target the IL-2 receptor in the treatment of relapsed or refractory cutaneous T-cell lymphoma and the first FDA-approved product for Citius Pharmaceuticals.

Accelerated approval of Fabhalta for treating primary IgA nephropathy was based on positive results from the Phase III APPLAUSE-IgAN study.

Clinical trials for Crexont demonstrated a substantial increase in "Good On" time, representing a period without troubling adverse effects in patients with Parkinson disease.

Partnership with Abbott for Simplera CGM will expand access to the advanced automated insulin delivery.

Voranigo is the first targeted therapy in almost 25 years to be approved by the FDA for grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery.

Expanded indication makes Fibryga the first and only on-demand, virus-inactivated, human plasma-derived fibrinogen concentrate approved for this indication.

Regulatory action marks the first engineered cell therapy approved for a solid tumor cancer in the United States.

Approval marks the first time an immuno-oncology regimen has demonstrated a survival benefit for all adults with primary advanced or recurrent endometrial cancer, company says.

Results from the PERSEUS study led to the FDA approval of Darzalex Faspro in patients with newly diagnosed multiple myeloma who are eligible for an autologous stem cell transplant.

With a growing number of legal battles over women’s health issues, such as abortion and infertility, stakeholders are concerned about the impact on everything from healthcare access to new technology.

Erzofri is the first patented paliperidone palmitate long-acting injectable developed in China to receive FDA approval for treating schizophrenia and schizoaffective disorder, company says.

Results from the ASC4FIRST study lead to FDA priority review designation of Scemblix in newly diagnosed patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase.

Approval of Zunveyl offers a novel approach with a dual mechanism of action designed to improve tolerability and efficacy in treating Alzheimer disease, company says.

Leqselvi is classified as a novel oral Janus Kinase inhibitor that targets JAK1 and JAK2 pathways, thought to be involved in the autoimmune response that leads to alopecia areata.

FDA’s Oncologic Drugs Advisory Committee acknowledged that Imfinzi met its primary endpoint of event-free survival in the treatment of resectable non-small cell lung cancer.

The study is expected to evaluate the safety of Iomab-ACT in sickle cell disease patients who are undergoing allogeneic bone marrow transplants.

The supplemental New Drug Application for brexpiprazole in combination with sertraline is supported by data from three randomized clinical trials that evaluated its safety and efficacy in adult patients with post-traumatic stress disorder.

Expanded indication of Brineura allows for treatment of neuronal ceroid lipofuscinosis type 2 disease in both symptomatic and presymptomatic children.

The new approval expands Xeomin’s indications to include horizontal forehead lines and lateral canthal lines.