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 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. For many years, systemic corticosteroids have been the mainstay of BP treatment.

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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. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis.

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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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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). mmHg (P=0.0003) In-office seated resting SBP decreased by −9.2

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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

Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] 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). 25 April 2023.

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New Drug Approvals

JULY 9, 2025

Sunvozertinib , sold under the brand name 舒沃哲, among others is an anti-cancer medication used for the treatment of non-small-cell lung cancer. [2] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]

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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). Those assigned to the active treatment arm will receive quarterly doses of ION582 at either 40 mg or 80 mg.

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Broad Institute

JULY 21, 2025

There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& The team used a scalable approach to develop prime editing treatments that directly repaired five different AHC-causing genetic mutations. The results really exceeded our expectations,” said Sakai. “It

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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] 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 November 2006.

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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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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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The Pharma Data

JULY 2, 2025

Despite its impact, there are currently no FDA-approved therapies for PMN , and treatment options are limited to non-specific and often toxic agents such as chemotherapy or general immunosuppressants. Despite the severity of PMN, nearly one-third of patients fail to achieve remission with current treatment strategies.

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The Pharma Data

JUNE 15, 2025

According to the data, the combination demonstrated a high ORR and a strong depth of response in a patient population with limited treatment options and poor outcomes under standard care. were penta-drug refractory, having been exposed to additional lines of treatment. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.

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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. Some of these treatments, like K.J.’s, They plan to harness scientific, medical, regulatory, and manufacturing innovations to enable on-demand genetic treatments like K.J.’s

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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 label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] Fitusiran 1711.0g/mol,

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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] 7] [12] In the European Union, pirtobrutinib is indicated for the treatment of mantle cell lymphoma. [2] 5] It is taken by mouth. [1] 2] PATENTS Guisot, N. 27 January 2023.

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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

Food and Drug Administration’s (FDA) accelerated approval of Lynozyfic™ (linvoseltamab-gcpt), a first-in-class BCMAxCD3 bispecific antibody, for the treatment of adults with relapsed or refractory (R/R) multiple myeloma (MM). Lynozyfic demonstrated early, deep, and durable responses in heavily pre-treated patients.

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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). For ambulatory patients — those retaining some ability to walk — there are no new treatment interruptions or regimen modifications at this time. 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

JUNE 29, 2025

Taletrectinib CAS 1505514-27-1 as salt: 1505515-69-4 , Taletrectinib adipate FDA 6/11/2025, Ibtrozi, To treat locally advanced or metastatic ROS1-positive non-small cell lung cancer AB-106, DS-6051a 405.5 1] Taletrectinib was approved for medical use in the United States in June 2025. [3] 1] Taletrectinib is a kinase inhibitor. [1]

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New Drug Approvals

APRIL 18, 2025

Formula C 27 H 29 Cl 2 FN 2 OS Crinecerfont , sold under the brand name Crenessity , is a medication used for the treatment of congenital adrenal hyperplasia. [1] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2]

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Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. These challenges have led to strategies such as directly observed therapy (DOT), in which nurses or physicians monitor patients to ensure they take their medicine every day.

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Codon

JUNE 5, 2025

.” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. Therapeutics The first Cas13-based clinical trial was approved in 2024.

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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. Importantly, the treatment effect emerged early in the course of therapy and continued to increase over time.

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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

It is being investigated as a potential treatment for various herpes infections, including those resistant to traditional antivirals like acyclovir. 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 Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 9] It is used as landiolol hydrochloride.

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Codon

NOVEMBER 1, 2024

Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease.

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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.

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FDA Law Blog: Drug Discovery

JUNE 18, 2025

As defined by the National Cancer Institute, tumor board meetings convene multidisciplinary specialists to decide on the best treatment plan for new and complex cancer cases. FDA plans to issue a limited number of vouchers, without specifying an exact number as of yet. The vouchers will be issued to “companies aligned with U.S.

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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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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). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.

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