Crown Bioscience

JULY 17, 2025

Both the novel and supplemental oncology approvals reflected recent developmental innovations, including an increased focus on targeted, immunologically driven, and personalized oncology therapies, targeting a broad range of cancers.

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The Pharma Data

JUNE 26, 2025

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.

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Fierce BioTech

JULY 15, 2025

For biotech companies advancing oncology therapies, regulatory speed can be critical to achieving key milestones and getting new therapies to patients faster. Regulatory expert shares how early planning and FDA engagement can speed oncology approval

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BioPharma Drive: Drug Pricing

MAY 27, 2025

Elsewhere, Angelini Pharma licensed a rare disease therapy and the FDA refused to review a Savara lung drug. City will receive $46 million from Biogen under the alliance.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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The Pharma Data

JUNE 25, 2025

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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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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Covalent Modifiers

MARCH 16, 2025

Here, we demonstrate the utility of a high-throughput in vitro screening platform along with a comprehensive panel to aid in the characterization of 15 Brutons tyrosine kinase (BTK) inhibitors that are either approved by the FDA or presently under clinical evaluation.

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Chemical Biology and Drug Design

JUNE 19, 2025

The presence of thienopyrimidine derivatives in several FDA-approved drugs and clinical trial candidates underscores their therapeutic potential and safety profile. As our understanding of their structure–function relationships deepens, we can expect further advancements in the development of thienopyrimidine-based therapies.

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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Drugs.com

MAY 5, 2025

Food and Drug Administration has approved the SAPIEN 3 platform, a transcatheter aortic valve replacement (TAVR) therapy, for patients with severe aortic stenosis without symptoms. MONDAY, May 5, 2025 -- The U.S. The approval is the first for TAVR.

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The Pharma Data

JUNE 24, 2025

This alliance aims to address mounting challenges in the development and commercialization of mAb therapies and gene therapies, particularly those involving AAV vectors. To date, more than 160 monoclonal antibody therapies targeting nearly 100 disease-related proteins have received regulatory approval globally.

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Drug Target Review

JULY 3, 2025

New approach methodologies data In April, the US Food and Drug Administration (FDA) announced a plan to replace animal testing in the development of monoclonal antibodies and other therapies with validated “human-relevant” methods, including AI-based computational models evaluating toxicity, cellular lines and organoid toxicity.

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New Drug Approvals

JULY 5, 2025

Combination Therapy: Palazestrant is being evaluated in combination with other drugs like CDK4/6 inhibitors (e.g., FDA Fast Track Designation: The FDA has granted Fast Track designation for the treatment of ER+/HER2- metastatic breast cancer that has progressed following endocrine therapy with a CDK4/6 inhibitor.

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The Pharma Data

JUNE 11, 2025

FDA Approves Tablet Formulation of BeOne’s BRUKINSA® for All Approved Indications, Offering Greater Convenience for Patients with B-cell Cancers BeOne Medicines Ltd. Food and Drug Administration (FDA). Notably, BRUKINSA has emerged as the market leader in new CLL patient starts across all lines of therapy in the U.S.

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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

JULY 9, 2025

1] Medical uses In the US, sunvozertinib is indicated for the treatment of adults with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. [1] 2 July 2025.

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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). A New Era in Infant RSV Protection Dr. Dean Y.

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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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New Drug Approvals

JUNE 24, 2025

FDA Designation: Nerandomilast received Breakthrough Therapy Designation from the FDA for the treatment of IPF. Progressive Fibrosing ILDs: Nerandomilast is also being investigated in other progressive fibrosing interstitial lung diseases (ILDs) beyond IPF.

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thought leadership

JUNE 18, 2025

An inside look at FDA's new Commissioner's National Priority Voucher (CNPV) pilot program—what it is, why it matters, and how regulatory and development teams can leverage it to accelerate high-priority therapies.

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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. This has underscored the urgent need for non-immunosuppressive, targeted therapies with more favorable safety profiles.

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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. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients aged seven years and older with narcolepsy.

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The Premier Consulting Blog

NOVEMBER 6, 2024

Table 1 : Examples of Common ICPs pMDI DPI Nebulizer Combination products are beneficial for respiratory treatments as they offer the potential to improve the efficacy and safety of therapies.

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SugarCone Biotech

JUNE 27, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. As we’ll see, ASCO 2025 was a breakout conference for B7-H3-targeted therapies. Advanced therapies targeting B7-H3 use different modalities, currently dominated by ADCs.

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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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BioPharma Drive: Drug Pricing

JULY 8, 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. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.

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The Pharma Data

JUNE 27, 2025

These data offer new hope to the CDD community, which has long struggled with a lack of effective seizure control therapies. The lack of approved therapies specifically indicated for CDD means that treatment strategies are often based on off-label use of anti-seizure medications, with limited success.

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Drug Target Review

JULY 7, 2025

But growing ethical scrutiny, supply shortages, cost burdens, scientific innovation and regulatory shifts like the US Food and Drug Administration (FDA)’s new alternative methods roadmap are bringing the continued reliance on live NHPs into question, and opening the door to next-generation solutions that could eventually replace them altogether.

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The Pharma Data

JUNE 18, 2025

The company’s lead candidate, NCX-L2 , is specifically designed to slow or even halt the progression of Parkinson’s disease—a neurodegenerative condition that currently has no disease-modifying therapies on the market. While current treatments like levodopa temporarily alleviate symptoms, none are capable of slowing or stopping neuronal loss.

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Broad Institute

JULY 21, 2025

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. The results really exceeded our expectations,” said Sakai. “It It was incredibly exciting to see that data.”

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Alta Sciences

FEBRUARY 19, 2025

Food and Drug Administration (FDA) issued two guidance documents outlining the necessary evaluations during the clinical development of oligonucleotide therapeutics: Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics and Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics .

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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. WuXi Biologics sets a new standard in the formulation of biologics by enabling concentration levels up to 230 mg/mL , a significant leap beyond the previous high mark of 200 mg/mL seen in FDA-approved products.

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New Drug Approvals

APRIL 25, 2025

3] Primary endocrine resistance was defined as relapse while on the first two years of adjuvant endocrine therapy (ET) and secondary endocrine resistance was defined as relapse while on adjuvant ET after at least two years or relapse within twelve months of completing adjuvant ET. [3] Food and Drug Administration (FDA).

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New Drug Approvals

MAY 17, 2025

4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [7] 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). 3 November 2006. January 2024.

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The Pharma Data

JUNE 6, 2025

Otsuka Unveils Promising Phase 3 Results for Sibeprenlimab in IgA Nephropathy, Marking Significant Proteinuria Reduction and Advancing a Novel APRIL-Targeted Therapy Otsuka Pharmaceutical Development & Commercialization, Inc., in those receiving placebo—further underscoring the tolerability of this investigational therapy.

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The Pharma Data

JUNE 12, 2025

This design was meant to enable clinician-researchers to gauge the ability of rilzabrutinib to control disease activity after the withdrawal of standard therapy. Fast Track and Orphan Drug Designations from FDA The U.S. Subsequently, rilzabrutinib was continued as monotherapy for the rest of the 52-week study period.

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