The Pharma Data

JULY 2, 2025

Biogen Launches Global Phase 3 PROMINENT Trial Evaluating Felzartamab in Primary Membranous Nephropathy Biogen Inc. The trial is expected to complete in 2029. This randomized, open-label study will compare felzartamab with tacrolimus, a commonly used immunosuppressant, in a cohort of approximately 180 patients worldwide.

Trials

The Pharma Data

JUNE 11, 2025

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We

Trials

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). According to Dr. Richard J.

FDA Approval

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] “Sunvozertinib: First Approval” Drugs. 12 (3): 217–224.

FDA

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.

FDA

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). “Biogen’s antisense ALS drug shows promise in early clinical trial” FierceBiotech. 25 April 2023.

FDA Approval

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.

FDA Approval

SugarCone Biotech

JUNE 19, 2025

Two large and randomized clinical trials, IMpower-133 and CASPIAN, showing statistically significant improvements in outcomes when chemotherapy was combined with atezolizumab or durvalumab, respectively, in the first-line treatment of ES-SCLC. This Phase 3 trial compared tarlatamab with chemotherapy as second-line treatment.

Therapies

Broad Institute

JULY 21, 2025

The technology has already been successfully tested in a clinical trial for another rare genetic disease. The scientists delivered the prime editors to cells in mice using clinically validated viruses called AAVs, which are already used in FDA-approved gene therapies targeting brain cells.

Disease

The Pharma Data

JUNE 25, 2025

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. These trials reflect Sanofi’s broad development program for riliprubart , which spans multiple rare and immune-mediated conditions.

FDA

New Drug Approvals

JULY 25, 2025

Syn EuropeanJournalofMedicinalChemistry265(2024)116124 Vamorolone (Agamree) On October 26, 2023, Vamorolone, developed jointly by Santhera Pharmaceuticals and ReveraGen BioPharma, has received FDA approval to treat DMD in patients aged 2 years and older [1]. Shale, U.J. 79 (2022) 1005–1014. [71] Reeves, E.P. Hoffman, K. Nagaraju, J.M.

FDA

New Drug Approvals

APRIL 25, 2025

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 19] Society and culture Legal status In October 2024, the US Food and Drug Administration (FDA) approved inavolisib for the treatment of PIK3CA -mutant breast cancer based on the results from the INAVO120 trial. [3] 3 November 2006.

FDA

New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] History The efficacy and safety of fitusiran were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male participants with either hemophilia A or hemophilia B. [2] Fitusiran 1711.0g/mol,

Clinical Trials

Broad Institute

MAY 21, 2025

In the early 2000s, both teams began preparing for clinical trials. In 2012, the University of Pennsylvania team and Novartis launched a collaboration to further develop the technology, which led to the FDA approval for the first commercial CAR T-cell therapy in 2017. That boosting signal is a staple of all CARs used today.

Therapies

Broad Institute

JUNE 24, 2025

Together, these technologies have entered at least 19 clinical trials, with clinical results from seven of those trials reported so far — all showing that the base edit or prime edit resulted in patient benefit. Of the 19 base editing and prime editing clinical trials underway now, more are taking place outside the U.S.

Treatment

New Drug Approvals

JUNE 11, 2025

The FDA rejected approval for gepirone in 2002 and 2004. [5] 5] It was submitted for the preregistration (NDA) phase again in May 2007 after adding additional information from clinical trials as the FDA required in 2009. 12] However, in March 2016, the FDA reversed its decision and gave gepirone ER a positive review. [13]

FDA

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

The Pharma Data

JUNE 15, 2025

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). The FDA agrees with this cautious approach. The study forms a key piece of the accelerated approval process for non-ambulatory patients in the United States. ELEVIDYS is currently the only U.S.

Immune Response

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] The FDA granted the application for elacestrant priority review and fast track designations. [2] Food and Drug Administration (FDA). 1] [4] It is taken by mouth. [1] PMC 9340905.

FDA

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] The trial was conducted at 49 sites in 10 countries in the United States, Europe, Australia, and Asia. [6] 6] The same trial was used to assess safety and efficacy. [6]

FDA Approval

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] Food and Drug Administration (FDA).

FDA

The Premier Consulting Blog

JANUARY 14, 2025

Project Facilitate A single point of contact call and information Help oncology healthcare providers or regulatory professionals with submitting an Expanded Access Request through FDAs Expanded Access Program for an individual cancer patient. Provides the Oncology Dosing Toolkit. Sponsors can request Project Orbis for their applications.

Clinical Trials

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.” Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.

Therapies

Codon

NOVEMBER 3, 2024

Still, while these developments provide cause for hope, each drug or vaccine faces a variety of challenges, ranging from financial incentives for clinical trials to the difficulty of discovering new antimicrobials that pass safety and efficacy tests. A phase 3 clinical trial for M72/AS01E, funded by the Gates Foundation, began this year.

Vaccine

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Dozens more clinical trials, based upon similar gene-editing technologies, are now underway. Cas9 is also being used in a number of ongoing clinical trials for everything from HIV to protein folding disease and lymphoma.

DNA

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.

FDA Approval

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] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2] 1 December 2024.

FDA

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 (FDA).

International

New Drug Approvals

JULY 10, 2025

3] Society and culture Legal status Sebetralstat was approved for medical use in the United States in July 2025. [1] Retrieved 9 July 2025. ^ “KalVista Pharmaceuticals Announces FDA Approval of Ekterly (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema” (Press release). 7 July 2025.

FDA

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. label of WINREVAIR based on compelling new evidence from the Phase 3 ZENITH trial. risk score of 9 or greater.

Trials

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.

FDA Approval

New Drug Approvals

JULY 8, 2025

Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. Clinical Trials: Pritelivir mesylate is currently under extensive study to evaluate its efficacy and safety profile, with promising results in early clinical trials. Johnston, C. 2016.18189.

Virus

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 Randomized clinical trials have been published to compare landiolol with placebo< [21] [22] [23] diltiazem, [24] and amiodaron [25] in patients with or without heart failure. AOP Health.

FDA

Codon

NOVEMBER 1, 2024

Although Amodei does acknowledge some real-world issues limiting scientific progress — such as the slow growth of organisms and tedious clinical trials — he mostly passes over the more general tools that will be required to accelerate research in the near term. This essay focuses on how we might do both, specifically for the cell.

DNA

BioPharma Drive: Drug Pricing

JULY 10, 2025

Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Moderna COVID vaccine gets full approval for children The approval comes amid regulatory upheaval under HHS head Robert F. TechTarget, Inc.s

Vaccine

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. Safety outcomes from the trial further bolster the confidence in MAVYRET’s use.

FDA Approval

Drug Patent Watch

JANUARY 7, 2025

Explain the FDA Approval Process Many patients are unaware of the rigorous approval process generic drugs must undergo. Educate them about the FDA’s role in ensuring the safety and efficacy of generic medications. These anecdotes can help alleviate fears and build confidence in generic medications.

Drugs