Drug Target Review

JUNE 5, 2025

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

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SugarCone Biotech

JUNE 19, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). This leads us to Amgen’s T cell-engager therapeutic, tarlatamab.

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The Pharma Data

JUNE 9, 2025

Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. It is also approved for adult patients with idiopathic hypersomnia (IH). Source link

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New Drug Approvals

JULY 9, 2025

2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]

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The Pharma Data

JUNE 25, 2025

FDA Orphan Drug Designation for Antibody-Mediated Rejection in Solid Organ Transplantation The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.

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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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The Pharma Data

JUNE 11, 2025

(Nasdaq: IONS) has announced the dosing of the first patient in the Phase 3 REVEAL clinical trial, marking a significant milestone in the development of ION582, an investigational therapy for Angelman syndrome (AS). Ionis Pharmaceuticals has a well-established track record in the development of antisense therapies for neurological disorders.

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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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The Pharma Data

JULY 2, 2025

Primary membranous nephropathy is a rare and serious autoimmune kidney disease characterized by the formation of autoantibodies that damage the glomerular basement membrane, often resulting in nephrotic syndrome —a condition marked by high levels of protein in the urine, severe swelling, and a significant risk of progression to kidney failure.

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Broad Institute

MAY 21, 2025

Merkin Prize in Biomedical Technology for developing chimeric antigen receptor (CAR) T-cell therapy, a groundbreaking form of personalized cancer immunotherapy that turns T cells into tumor killers and has led to durable remissions in tens of thousands of patients with previously incurable blood cancers.

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Broad Institute

JUNE 24, 2025

Muldoon, a baby boy born without the ability to process dietary protein properly, had become the first person to be treated with a customized gene editing therapy. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases. The team that treated K.J. The result appears to be a major success.

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The Pharma Data

JUNE 15, 2025

The study, which is the largest to date that focuses exclusively on this patient population, shows high overall response rates (ORR) alongside deep and durable responses, offering much-needed hope for individuals whose disease typically carries a poor prognosis. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.

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New Drug Approvals

MAY 9, 2025

2] Elacestrant was approved for medical use in the United States in January 2023, [1] [2] [5] [6] and in the European Union in September 2023. [3] 2] Participants were randomly assigned in a 1:1 ratio to receive either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy. 8 February 2023.

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FDA Pharmacokinetics Therapies Treatment 62

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.

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The Pharma Data

JULY 2, 2025

This accelerated pathway reflects the FDA’s recognition of an urgent medical need in advanced-stage multiple myeloma and is based on early clinical indicators such as objective response rate (ORR) and durability of response (DoR). Yancopoulos, President and Chief Scientific Officer of Regeneron. In the U.S.

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The Pharma Data

JUNE 15, 2025

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). The ultimate aim is to enable the administration of ELEVIDYS safely while honoring its potential to provide a dramatic and durable therapeutic benefit for children battling this profoundly challenging disease.

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Immune Response Regulations Therapies Disease 40

The Premier Consulting Blog

JANUARY 14, 2025

Established in 2017 under the 21st Century Cures Act, the OCE brings together multidisciplinary scientific expertise to accelerate the review and approval of drugs, biologics, and medical devices for cancer care. These reviews have provided life-saving cancer therapies a median of 3.1 Provides the Oncology Dosing Toolkit.

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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] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 28 March 2025.

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New Drug Approvals

MAY 15, 2025

5] Leniolisib was approved for medical use in the United States in March 2023. [5] 5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] 5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] “Leniolisib: First Approval” Drugs.

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Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.”

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DNA RNA Clinical Trials Engineering 81

New Drug Approvals

JUNE 29, 2025

1] [2] Adverse effects The FDA prescribing information for taletrectinib includes warnings and precautions for hepatotoxicity, interstitial lung disease/pneumonitis, QTc interval prolongation, hyperuricemia, myalgia with creatine phosphokinase elevation, skeletal fractures, and embryo-fetal toxicity. [1] 26 (6): 765–772. PMID 40170301.

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International FDA Clinical Trials Treatment 62

New Drug Approvals

APRIL 18, 2025

2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2] Food and Drug Administration (FDA) (Press release).

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The Pharma Data

JULY 2, 2025

Importantly, the treatment effect emerged early in the course of therapy and continued to increase over time. Despite existing therapies, many PAH patients remain at substantial risk. Patients were randomized 1:1 to receive either WINREVAIR or placebo, both in combination with maximum-tolerated background therapy.

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Trials FDA Licensing Licensing 52

The Pharma Data

JUNE 13, 2025

FDA Approves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S. Subsequently, KEYTRUDA is used as monotherapy following the completion of adjuvant therapy.

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New Drug Approvals

JULY 8, 2025

US FDA granted fast track designation for pritelivir in 2017 and breakthrough therapy designation 2020. HSV in immunocompromised patients Although HSV infection is very common in the general population, it rarely causes serious disease and is effectively contained by the immune system. References ^ Wilck, M.B.; Zuckerman, R.A.;

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Virus DNA Clinical Trials Treatment 62

BioPharma Drive: Drug Pricing

JULY 10, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. The Centers for Disease Control and Prevention currently recommends shared clinical decision-making for healthy children.

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The Pharma Data

JUNE 11, 2025

FDA Expands Approval of AbbVie’s MAVYRET® as First and Only 8-Week Treatment for Acute Hepatitis C in Adults and Children Aged 3 and Above AbbVie has received a significant regulatory boost for its hepatitis C treatment portfolio as the U.S. Global and U.S. Safety outcomes from the trial further bolster the confidence in MAVYRET’s use.

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The Pharma Data

JUNE 24, 2025

Regeneron Unveils Groundbreaking $200 Million Donation Matching Program to Expand Patient Access to Vision-Saving Treatments Regeneron Pharmaceuticals , a leader in biotechnology and a longtime advocate for equitable healthcare access, has announced a bold new philanthropic initiative designed to support patients facing debilitating eye diseases.

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BioPharma Drive: Drug Pricing

MAY 30, 2025

Keros will also stop work on a cardiovascular disease drug. Elsewhere, Stealth Biotherapeutics and Taysha Gene Therapies each think they've found a path to FDA approval.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

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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.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Muscle makes up about 40 percent of body weight. million DNA bases.

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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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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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Chemical Biology and Drug Design

OCTOBER 10, 2023

Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Molecular insights of HIV Reverse transcriptase and it's inhibitors.

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