BioPharma Drive: Drug Pricing

NOVEMBER 11, 2024

Developer Autolus set a list price of $525,000 for its new cell therapy, which it will sell for a type of leukemia under the brand name Aucatzyl.

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BioPharma Drive: Drug Pricing

JUNE 4, 2024

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

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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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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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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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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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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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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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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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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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BioPharma Drive: Drug Pricing

AUGUST 2, 2023

The company is discussing various payment models for its sickle cell treatment with government and commercial insurers ahead of an FDA approval decision later this year.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Results from the study that led to the FDA approval appeared in The Lancet Neurology in April 2024 with commentary. Researchers have been working on developing gene therapy for DMD for decades.

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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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BioPharma Drive: Drug Pricing

FEBRUARY 20, 2024

The FDA approved the targeted therapy together with chemo for first-line metastatic lung cancer, while fresh trial results supported earlier use.

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Covalent Modifiers

FEBRUARY 15, 2024

Recently, afatinib, an FDA-approved kinase inhibitor, has been shown to modulate brachyury levels in multiple chordoma cell lines. In doing so, we demonstrate the feasibility of direct brachyury modulation, which may further be developed into more potent tool compounds and therapies.

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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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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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The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., In the U.S.,

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FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). All the data described above can be found in Sections 12 and 14 of the FDA-approved prescribing information (see here ).

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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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New Drug Approvals

MAY 17, 2025

Jump up to: a b c d e f g h i j k l “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). Food and Drug Administration (FDA). Jump up to: a b New Drug Therapy Approvals 2023 (PDF). Food and Drug Administration (FDA) (Report).

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Broad Institute

JULY 19, 2023

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

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SCIENMAG: Medicine & Health

JULY 26, 2023

Food and Drug Administration (FDA) approval for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need […] PARAMUS, N.J.,

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New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants. [2] 2] The US Food and Drug Administration (FDA) granted the application for fitusiran orphan drug and fast track designations. .

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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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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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New Drug Approvals

MAY 8, 2025

7] Pirtobrutinib was approved for medical use in the United States in January 2023, [4] [8] [9] [10] and in the European Union in November 2023. [2] 4] Participants had a median of three prior lines of therapy, with 93% having two or more prior lines. [4] 5] It is taken by mouth. [1] 2] PATENTS Guisot, N. 27 January 2023. CCR-23-1272.

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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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ASPET

OCTOBER 12, 2023

The CET RAIDR program minimizes development and procurement costs by supplementing the military medical providers' toolbox with post-Phase II therapies that demonstrate established safety and manufacturing processes, leading to a cost-sparing model for niche medicines (i.e., CBRN MCMs).

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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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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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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.,

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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. The test is FDA-approved to report short variants in 311 genes including rearrangements and copy number losses in BRCA1 and BRCA2 genes.

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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%.

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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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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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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%

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