The Pharma Data

JUNE 20, 2025

FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. If approved in those regions, the therapy could become a global standard of care for this devastating disease.

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The Pharma Data

JUNE 9, 2025

FDA Approves Merck’s ENFLONSIA™ to Protect Infants from Severe RSV Illness Merck operating as MSD outside the United States and Canada, has received a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting approval for ENFLONSIA™ (clesrovimab-cfor).

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The Pharma Data

JUNE 25, 2025

FDA Approves Label Update for Lilly’s Amyvid, Expanding Its Role in Alzheimer’s Disease Diagnosis and Therapy Guidance In a major development for Alzheimer’s diagnostics, Eli Lilly and Company (NYSE: LLY) announced that the U.S.

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The Pharma Data

JUNE 16, 2025

FDA Approves ANDEMBRY (garadacimab-gxii) The First Once-Monthly Prophylactic HAE Therapy Targeting Factor XIIa CSL a leading biotechnology company with a strong track record of developing innovative medicines for patients with rare and serious disorders, today announced that the U.S. when directly comparing ANDEMBRY to placebo.

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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. The announcement is particularly relevant given the lack of FDA-approved treatment options for AMR, a complication that poses a critical threat to transplant success and long-term graft survival.

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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. SCLC generally has a “hot” tumor microenvironment (TME) meaning that it is infiltrated by immune cells, including the T cells that are activated by immune checkpoint therapies.

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The Pharma Data

JULY 2, 2025

Currently, more than 20% of FDA-approved monoclonal antibodies (mAbs) fall into this category, reflecting a growing industry trend. Moreover, these factors may affect drug safety and efficacy by increasing the likelihood of immunogenic responses. WuXiHigh™ 2.0: Pushing the Boundaries With WuXiHigh™ 2.0, Source link

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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).

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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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New Drug Approvals

MAY 17, 2025

3] Tofersen was approved for medical use in the United States in April 2023, [3] [6] and in the European Union in May 2024. [4] 4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [7] gmol 1 References ^ “Register of Innovative Drugs” Health Canada. 3 November 2006.

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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

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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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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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. wasn’t the first patient to receive base editing therapy. He needed a personalized, one-of-a-kind therapy. This unprecedented feat required diagnosing K.J.’s

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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. Retrieved 11 February 2023.

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New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 2] [6] Suzetrigine is the first medication to be approved by the US Food and Drug Administration (FDA) in this new class of pain management medicines. [2] Suzetrigine is taken by mouth. [1]

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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] Jump up to: a b c “Drug Trials Snapshots: Jaypirca” U.S.

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The Pharma Data

JUNE 15, 2025

The RedirecTT-1 trial shows the power of this novel combination approach as a potential treatment option for these patients, many of whom have limited options after multiple lines of therapy.” were penta-drug refractory, having been exposed to additional lines of treatment. Among these: 84.4% Among these: 84.4% Furthermore, 64.1%

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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] 26 March 2025.

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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

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The Pharma Data

JULY 2, 2025

FDA Grants Accelerated Approval to Regeneron’s Lynozyfic™ (linvoseltamab-gcpt) for Relapsed or Refractory Multiple Myeloma Regeneron Pharmaceuticals has secured a critical milestone in oncology drug development with the U.S. Yancopoulos, President and Chief Scientific Officer of Regeneron.

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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). With more than 900 individuals treated with ELEVIDYS to date, there’s a strong understanding within the community that many view this therapy as a beacon of hope. ELEVIDYS is currently the only U.S.

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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] New Drug Therapy Approvals 2023 (PDF).

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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).

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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] Food and Drug Administration.

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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. Such off-target cuts are obviously a big concern when developing human therapies, because they can cause mutations that lead to cancer or other diseases.

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The Pharma Data

JULY 2, 2025

FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025.

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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

1428321-10-1 Pritelivir mesylate is an antiviral drug currently under development, specifically targeting herpes simplex virus types 1 and 2 (HSV-1 and HSV-2). Pritelivir (development codes AIC316 or BAY 57-1293 ) is a direct-acting antiviral drug in development for the treatment of herpes simplex virus infections (HSV).

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New Drug Approvals

APRIL 19, 2025

Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 Mode of action The drug acts as an ultra-short-acting 1-selective blocking agent. Jump up to: a b “Novel Drug Approvals for 2024” U.S. 20 November 2023. AOP Health.

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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. This panel, known as ACIP for short, recommends who should receive FDA-approved vaccines.

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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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FDA Law Blog: Drug Discovery

JUNE 18, 2025

Livornese — After teasing a new rapid review pilot program for the past few weeks, on June 17, 2025, FDA officially announced the Commissioner’s National Priority Voucher (“CNPV”) program to expedite new drug and biologic (but not device or drug-device combination product) reviews. Addressing unmet public health needs.

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The Pharma Data

JUNE 24, 2025

While effective treatments do exist—many of them involving advanced biologic therapies that can slow, halt, or even reverse the progression of these conditions—the cost of care can remain a formidable barrier for many patients, particularly those on fixed or limited incomes. Food and Drug Administration (FDA).

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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

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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