FDA Approval Regulations Therapies 
FDA Law Blog: Drug Discovery
NOVEMBER 10, 2024
We note that the only Phase 3 study funded in the FY2024 announcement is the first-ever late-stage clinical trial for the indication of microcystic lymphatic malformations , a serious, rare genetic skin disease with no FDA-approved therapies. Relative to other areas of medicine (e.g.,
FDA Law Blog: Drug Discovery
DECEMBER 7, 2022
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
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The Pharma Data
APRIL 19, 2022
Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.
The Pharma Data
SEPTEMBER 7, 2020
Genentech’s once-daily oral therapy Gavreto (pralsetinib) has secured FDA backing in the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), it has emerged. In 27 treatment-naïve NSCLC patients, the ORR was found to be 70% and CRR was 11%.
The Pharma Data
AUGUST 13, 2020
NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. If this benefit cannot be proven, the FDA may withdraw its authorisation. . of normal at baseline to 5.9%
The Pharma Data
NOVEMBER 27, 2020
FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. The FDA granted approval under the accelerated approval regulation. NEW YORK, Nov.
New Drug Approvals
APRIL 25, 2025
3] Inavolisib was approved for medical use in the United States in October 2024. [3] 8] Therefore, it may serve as a new addition to combination therapy with conventional cancer treatment, such as chemotherapy. 3] [6] [20] [21] The drug application was granted priority review and breakthrough therapy designations by the FDA. [3]
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