PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done.

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Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

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BioPharma Drive: Drug Pricing

JUNE 29, 2023

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

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Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

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BioPharma Drive: Drug Pricing

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

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Drugs.com

JUNE 30, 2023

Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. FRIDAY, June 30, 2023 -- The U.S. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million for a single.

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The Pharma Data

DECEMBER 21, 2020

FDA Approves First Oral Hormone Therapy for Advanced Prostate Cancer. 21, 2020 — Orgovyx (relugolix) is now approved to treat advanced prostate cancer and is the first oral hormone therapy approved for this indication, the U.S. Professional. MONDAY, Dec. Food and Drug Administration announced Friday.

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The Pharma Data

MAY 27, 2022

Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

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BioPharma Drive: Drug Pricing

AUGUST 14, 2023

Elrexfio is the third bispecific antibody cleared to treat the blood cancer, and will compete with other therapies that target the “BCMA” protein on myeloma cells.

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Drugs.com

FEBRUARY 19, 2024

Food and Drug Administration has approved a novel treatment for advanced melanoma, the most deadly form of skin cancer. becomes the first cellular therapy approved to. Amtagvi, made by Iovance Biotherapeutics Inc.,

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The Pharma Data

AUGUST 9, 2021

deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease.

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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.

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Drugs.com

FEBRUARY 19, 2024

Food and Drug Administration has approved a novel treatment for advanced melanoma, the most deadly form of skin cancer.Amtagvi, made by Iovance Biotherapeutics Inc., becomes the first cellular therapy approved to.

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BioPharma Drive: Drug Pricing

JANUARY 5, 2024

recently submitted its MDMA capsules for FDA approval, following two positive studies. Lykos Therapeutics, formerly known as MAPS Public Benefit Corp.,

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The Pharma Data

AUGUST 19, 2021

Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel, advancing the company’s commitment to personalised healthcare through tests that determine which patients are most likely to benefit from specific and targeted therapies. today announced U.S. A bout the VENTANA MMR RxDx Panel.

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BioPharma Drive: Drug Pricing

JUNE 30, 2023

The agency granted a long-awaited clearance on Thursday, but unexpected aspects of the hemophilia treatment’s label had some investors worried about its commercial prospects.

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The Pharma Data

DECEMBER 17, 2020

Food and Drug Administration (FDA) had several approvals this week. Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy. The FDA approval of Klisyri is a significant milestone for Athenex.

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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

JANUARY 18, 2021

“It’s important that Impulse Dynamics acted quickly in a joint effort with the FDA to remove this potential barrier standing in the way of critical diagnostic procedures for a subset of patients with HF. CCM therapy is the first approach designed to optimize heart contraction, allowing more oxygen-rich blood to reach the body.

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Fierce BioTech

OCTOBER 5, 2023

Discover Strategies for Combatting Disruptions in Gene Therapy Development Cell and gene therapy development has exploded, with Q4 2022 showing more FDA approvals than over the past five years combined.[1]

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Drugs.com

JUNE 30, 2023

Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder. FRIDAY, June 30, 2023 -- The U.S. The treatment is not cheap: Roctavian will.

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The Pharma Data

JANUARY 17, 2021

Food and Drug Administration (FDA) approved Janssen Pharmaceuticals ’ (a Johnson and Johnson company) Darzalex Faspro for adults with newly diagnosed light chain amyloidosis. It was approved in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd). Michael Vi/Shutterstock. It was developed with Genmab.

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ASPET

JANUARY 31, 2024

Despite the standard of care therapy which includes surgical resection, temozolomide chemotherapy, radiation and the newly added tumor treating fields, median survival remains only ~20 months. Glioblastoma (GBM) is the most frequently diagnosed primary CNS tumor in adults.

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV. Air Force photo by Kemberly Groue.

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Chemical Biology and Drug Design

OCTOBER 10, 2023

Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Zidovudine, didanosine, and stavudine are FDA-approved NRTIs, while nevirapine, efavirenz, and delavirdine are FDA-approved NNRTIs.

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The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) gave a greenlight for ViiV Healthcare ’s Cabenuva. Today’s FDA approval of Cabenuva represents a shift in the way HIV is treated, offering people living with HIV a completely new approach to care,” said Lynn Baxter, Head of North America, ViiV Healthcare. clinical practices. “Not

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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%. Matt Fellows. Source link.

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BioPharma Drive: Drug Pricing

SEPTEMBER 5, 2023

The FDA-approved therapy, called Palforzia, was little used, leading Nestle to abandon a business it had secured in a $2.6 billion deal three years ago.

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The Pharma Data

JUNE 22, 2023

FDA approval Pfizer (NYSE: PFE) announced that the U.S. 3,4,5,6) “Despite treatment advancement in metastatic castration-resistant prostate cancer, the disease can progress quickly, and many patients may only receive one line of therapy. Pfizer’s TALZENNA® in combination with XTANDI® receives U.S.

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The Pharma Data

AUGUST 31, 2021

today announced a label update for KEYTRUDA, Merck’s anti-PD-1 therapy, for its indication in first-line advanced urothelial carcinoma (bladder cancer) in the U.S. Food and Drug Administration (FDA) has converted this indication from an accelerated to a full (regular) approval. Source link: [link].

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The Pharma Data

JANUARY 22, 2021

This approval will allow some patients the option of receiving once-monthly injections in lieu of a daily oral treatment regimen,” said Dr. John Farley, director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research.

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The Pharma Data

MAY 19, 2023

ABBOTT RECEIVES FDA APPROVAL FOR TACTIFLEX™ ABLATION CATHETER FOR TREATMENT OF ABNORMAL HEART RHYTHM Abbott (NYSE: ABT) today announced that the U.S. 6 “Abbott is leading the way in helping doctors manage common arrhythmias with the most holistic portfolio for this condition in the world,” said Christopher Piorkowski, M.D.,

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The Pharma Data

AUGUST 27, 2020

Roche is committed to improve patient outcomes by providing multiple testing options that support decision-making during all lines of therapy. Food and Drug Administration (FDA) has approved FoundationOne®Liquid CDx, Foundation Medicine’s comprehensive pan-tumour liquid biopsy test for patients with solid tumours.

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The Pharma Data

OCTOBER 8, 2021

Rethymic is the first thymus tissue product approved in the U.S. “Today’s action marks the first FDA approval of a therapy to treat this very rare and devastating disease in children,” said Peter Marks, M.D., director of the FDA’s Center for Biologics Evaluation and Research.

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The Pharma Data

NOVEMBER 29, 2021

Conventional treatment for ovarian cancer includes surgery to remove as many of the tumors as possible, chemotherapy to stop the growth of malignant cells or other targeted therapy to identify and attack specific cancer cells. The FDA previously granted Cytalux orphan-drug , priority and fast track designations. Related Information.

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The Premier Consulting Blog

NOVEMBER 12, 2024

For drug developers the 505(b)(2) pathway presents an expedited pathway to FDA approval. In our experience, the conversation on 505(b)(2) pathways commonly focuses on predicate data or bridging strategies to accelerate FDA approval but lacks dialogue on the needs of US payers. This is standard formulary policy language.

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