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

FDA Approval 40

FDA Approval FDA Pharmaceuticals Treatment 40

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.

FDA Approval 62

FDA Approval FDA Treatment Clinical Trials 62

SugarCone Biotech

JUNE 19, 2025

Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. months (versus 20% ORR and 8.3

Therapies 78

Therapies Clinical Trials Treatment Clinical Development 78

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. receiving placebo.

FDA Approval 40

FDA Approval Therapies FDA Disease 40

The Pharma Data

JUNE 25, 2025

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.

FDA 40

FDA FDA Approval Therapies Drugs 40

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]

FDA 62

FDA FDA Approval Clinical Trials Trials 62

thought leadership

FEBRUARY 26, 2025

Rare diseases are defined in the Orphan Drug Act as diseases or conditions that affect less than 200,000 people in the United States. Evaluating and developing treatments for rare diseases remains a key priority for FDA and is incentivized through the Orphan Drug Act.

Clinical Development 52

Clinical Development Disease Production FDA Approval 52

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.

Disease 141

Disease Laboratories Treatment DNA 141

The Pharma Data

JUNE 11, 2025

The study represents a major step toward addressing the unmet medical needs of individuals affected by this rare and severe neurodevelopmental disorder, which currently has no approved disease-modifying treatments. Those assigned to the active treatment arm will receive quarterly doses of ION582 at either 40 mg or 80 mg.

Trials 40

Trials Therapies Treatment Disease 40

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.

Trials 40

Trials Disease FDA Approval Treatment 40

Broad Institute

JUNE 24, 2025

Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. Some of these treatments, like K.J.’s, The team that treated K.J. Today, K.J.

Treatment 133

Treatment Disease DNA Clinical Trials 133

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.

FDA 57

FDA FDA Approval Therapies Trials 57

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.

Therapies 40

Therapies Treatment Disease FDA Approval 40

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.

Clinical Trials 107

Clinical Trials FDA FDA Approval Trials 107

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] Randomization was stratified based on whether the ESR1 mutation was detected or not, prior treatment with fulvestrant, and presence of visceral metastasis. [2] 1] [4] It is taken by mouth.

FDA 62

FDA Pharmacokinetics Therapies Treatment 62

Broad Institute

MAY 21, 2025

The $400,000 prize, to be shared among the four winners, recognizes their role in creating and advancing a technology that has reshaped how physicians treat leukemia, lymphoma, and multiple myeloma, and is now showing promise in treating autoimmune and infectious diseases. A single dose of the engineered cells wiped out signs of disease.

Therapies 119

Therapies FDA Approval Engineering Immune Response 119

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.

FDA 40

FDA FDA Approval Therapies Treatment 40

New Drug Approvals

MAY 8, 2025

1] [11] In December 2023, the US Food and Drug Administration (FDA) expanded the indication for pirtobrutinib to include the treatment of adults with chronic lymphocytic leukemia or small lymphocytic leukemia. [7] 7] [12] In the European Union, pirtobrutinib is indicated for the treatment of mantle cell lymphoma. [2]

FDA Approval 52

FDA Approval FDA Therapies Trials 52

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.

Immune Response 40

Immune Response Regulations Therapies Disease 40

The Pharma Data

JULY 3, 2025

a biotechnology company pioneering treatments based on human glycobiology, has announced significant leadership changes as the company accelerates its clinical and strategic initiatives. Synedgen Appoints Dr. Laura Saward as CEO and Dr. Kenton Gregory as CMO Amid Strategic Growth Synedgen, Inc., to President and Chief Executive Officer.

FDA Approval 40

FDA Approval Government FDA Engineering 40

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] hdl : 10665/330984. 24 March 2023.

FDA Approval 62

FDA Approval FDA Treatment International 62

The Premier Consulting Blog

JANUARY 14, 2025

The MORE team provides a unified clinical review of the program, while the product center makes the final approval determination, considering both clinical and non-clinical information holistically. These include Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation, and the Accelerated Approval Pathway (AAP).

Clinical Trials 52

Clinical Trials Therapies FDA Trials 52

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]

International 62

International FDA Clinical Trials Treatment 62

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]

FDA 62

FDA FDA Approval Drugs Trials 62

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. Dozens more clinical trials, based upon similar gene-editing technologies, are now underway.

DNA 81

DNA RNA Clinical Trials Engineering 81

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. Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. million people every year.

Vaccine 113

Vaccine Trials Therapies Treatment 113

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).

Virus 62

Virus DNA Clinical Trials Treatment 62

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.

Trials 52

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.

FDA Approval 40

FDA Approval Treatment FDA Therapies 40

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. This essay focuses on how we might do both, specifically for the cell.

DNA 132

DNA RNA Molecular Biology Laboratories 132

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.

FDA Approval 40

FDA Approval FDA Virus Therapies 40

The Pharma Data

JUNE 16, 2025

Lilly to Expand Access to Zepbound: All Approved Doses Now Available in Vials Through LillyDirect’s Self-Pay Pharmacy Solutions Eli Lilly and Company today made a significant announcement aimed at improving patient access to its obesity treatment Zepbound (tirzepatide). Importantly, all Zepbound single-dose vials — from the 2.5

Pharmacy 40

Pharmacy FDA Approval Treatment Disease 40

Drug Patent Watch

JANUARY 7, 2025

As healthcare professionals, it’s our responsibility to educate patients about generic drugs and empower them to make informed decisions about their treatment options. Explain the FDA Approval Process Many patients are unaware of the rigorous approval process generic drugs must undergo. World Health Organization.

Drugs 76

Drugs FDA Doctors Trials 76

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.

Treatment 40

Treatment Disease Therapies FDA Approval 40

Drugs.com

FEBRUARY 6, 2025

Food and Drug Administration has approved Onapgo (apomorphine hydrochloride) injection as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in patients with. THURSDAY, Feb. 6, 2025 -- The U.S.

FDA Approval 263

FDA Approval Disease FDA Treatment 263

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.

FDA Approval 313

FDA Approval Therapies FDA Treatment 313

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.

FDA Approval 246

FDA Approval Therapies FDA Treatment 246