FDA Law Blog: Drug Discovery

JANUARY 30, 2025

To this end, the December 2024 draft guidance and a companion guidance published January 7, 2025, Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway (the January 2025 draft guidance ) describe FDAs latest thinking on what it means to conduct a confirmatory trial with due diligence.

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The Pharma Data

JUNE 18, 2025

Garg will oversee a newly integrated research and development (R&D) organization designed to enhance innovation, expedite drug development, and better address unmet medical needs through tighter alignment of scientific discovery and clinical execution.

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SugarCone Biotech

JUNE 27, 2025

A post-ASCO update on tumor targets DLL3, B7H3 and Her3 June 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and Her3 are compelling tumor antigens to target with cancer therapeutics. As we’ll see, ASCO 2025 was a breakout conference for B7-H3-targeted therapies.

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Drug Target Review

JUNE 5, 2025

Dr Aaron Haubner, Senior Manager of North America Medical Affairs and Market Access at Terumo Blood and Cell Technologies , reveals that while promising new treatments emerge, urgent partnerships are needed to ensure this essential blood therapy reaches the patients who need it most.

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Drug Target Review

JUNE 16, 2025

By complementing traditional analytical methods, it provides a more accurate representation of drug behaviour to support translational and pre-clinical studies. This article explores the principles, applications and future potential of MSI in drug development. Drug Discovery Today 2025, 30 (1), 104275.

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Alta Sciences

MARCH 4, 2025

Forging a Path Forward for Substance Abuse Potential Studies With the Cross-Company Abuse Liability Council By Beatrice Setnik, PhD pmjackson Tue, 03/04/2025 - 20:10 Many of my colleagues and I are members of the Cross-Company Abuse Liability Council (CCALC), where I have been a co-chair for the past seven years, and a member for 15.

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The Pharma Data

JUNE 9, 2025

The COACH study is the first clinical investigation to assess the combined effects of two once-weekly treatments—TransCon® CNP (navepegritide) and TransCon® hGH (lonapegsomatropin)—in children with achondroplasia. TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia,” Dr. Shu stated.

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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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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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BioPharma Drive: Drug Pricing

JULY 14, 2025

Published July 14, 2025 Ben Fidler Senior Editor post share post print email license Takeda reported positive results for oveporexton, a drug being developed for narcolepsy Type 1, on July 14, 2025. and elsewhere in its 2025 fiscal year and plans to present detailed results at an upcoming medical meeting.

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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. Patient advocacy groups also welcomed the development.

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The Pharma Data

JUNE 26, 2025

BeOne Medicines Showcases Bold Vision and Expansive Oncology Pipeline at 2025 R&D Day BeOne Medicines Ltd., a global oncology-focused biopharmaceutical company, delivered a comprehensive update to investors today during its highly anticipated Research and Development (R&D) Day.

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SugarCone Biotech

JULY 6, 2025

A post-ASCO update on tumor targets DLL3, B7H3 and HER3 July 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and HER3 are compelling tumor antigens to target with cancer therapeutics. This is an active drug development landscape with a lot of recent news. regulatory application in NSCLC.

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BioPharma Drive: Drug Pricing

JUNE 11, 2025

Published June 11, 2025 Ben Fidler Senior Editor post share post print email license The FDA on June 11, 2025 approved Nuvation's Ibtrozi for ROS1-positive non-small cell lung cancer. Sanofi’s $9.1 Track the deals that are happening here. Sanofi’s $9.1

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Alta Sciences

APRIL 24, 2025

Case Study: How Altasciences Overcame Pharmacological Challenges in a GLP-1 IND-Enabling Study pmjackson Thu, 04/24/2025 - 21:43 For emerging therapies with pronounced pharmacological effects, thoughtful study design is critical to ensuring reliable and interpretable data. This blog was originally published in April 2025.

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BioPharma Drive: Drug Pricing

JUNE 26, 2025

Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease. Vor Biopharma licensed a drug in June 2025 that targets proteins essential to B cell survival. But a rocky few years forced Vor to change direction.

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BioPharma Drive: Drug Pricing

JULY 10, 2025

Published July 10, 2025 Ben Fidler Senior Editor post share post print email license Soleno Therapeutics on July 10, 2025 announced preliminary sales for its new Prader-Willi Syndrome drug Vykat. By Jonathan Gardner • Updated April 9, 2025 Keep up with the story.

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BioPharma Drive: Drug Pricing

JULY 2, 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 Organon drug for endometriosis falls short in mid-stage study Company executives had seen the drug as a potential multi-billion dollar opportunity in women’s health.

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BioPharma Drive: Drug Pricing

JULY 9, 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 Merck to buy Verona and its lung drug in $10B deal The New Jersey-based pharma has inked one of the biggest deals of the year in pursuit of a promising new way to treat COPD.

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FDA Law Blog: Drug Discovery

FEBRUARY 11, 2025

On February 5, 2025, a press release caught our eye Arbor Biotechnologies announced that ABO-101, its investigational therapy for the treatment of primary hyperoxaluria type 1 (PH1), received a rare pediatric disease designation.

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The Pharma Data

JUNE 6, 2025

Therapeutic Specialization: Where CSOs Make the Greatest Impact Therapeutic areas characterized by complex treatment protocols , specialist physician audiences , and dynamic clinical pipelines are ideal candidates for CSO deployment. These include: Oncology : Due to rapid innovation, evolving standards of care, and high unmet needs.

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BioPharma Drive: Drug Pricing

JUNE 27, 2025

Published June 27, 2025 Ned Pagliarulo Lead Editor post share post print email license Bristol Myers Squibb sells two CAR-T cell therapies for cancer. On June 26, 2025, the Food and Drug Administration eased some of the medicines' reporting requirements.

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Conversations in Drug Development Trends

APRIL 29, 2025

Unfortunately, this years event, scheduled for February 27-28, 2025, in Washington, DC, was abruptly postponed less than one week before it began. Thats why Worldwide Clinical Trials was committed to ensuring the rare disease community still had a space to share knowledge, resources, and support on Rare Disease Day 2025.

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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. Tobolowsky & Michelle L.

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Codon

JULY 14, 2025

Salicylic acid is likely the active compound that blocks the enzyme cyclooxygenase , giving aspirin its efficacy, and was used as a treatment for fever and gout before aspirin was developed. He treated fifty people over five years, all of whom he said were either cured or helped by the treatment. Credit: Wellcome Collection.

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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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Perficient: Drug Development

FEBRUARY 28, 2025

The 2025 National Conference on Artificial Intelligence is an unparalleled opportunity to dive deep into the transformative potential of AI across various sectors. With topics ranging from responsible AI to workforce development, this conference explores the vast possibilities of AI.

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Conversations in Drug Development Trends

APRIL 28, 2025

Following the American College of Cardiology (ACC) conference 2025, Worldwide Clinical Trials hosted a post-meeting webinar, On the Cutting Edge of Cardiovascular Discovery Learnings from ACC 2025 and Future Perspectives. Ziecina began the session by reflecting on the wealth of new data revealed at ACC 2025.

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BioPharma Drive: Drug Pricing

JUNE 23, 2025

Supply chain and ethical considerations The podcast discussion highlighted how soaring demand has strained manufacturing capacity, with contract manufacturing organizations dedicating large portions of their output to GLP-1 drugs. By Jacob Bell • May 6, 2025 Keep up with the story.

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Quanticate

SEPTEMBER 13, 2024

With the implementation of technology rapidly increasing, stricter regulatory standards, and growing demand for innovative treatments, the trends within the industry continue to evolve, impacting the future of clinical trials both positively and negatively.

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H1 Blog

DECEMBER 11, 2024

In this edition, our CEO and co-founder, Ariel Katz, along with Regional VP of Trial Landscape, Ryan Brown, share their top predictions for 2025. Drug Policy There will be significant changes in clinical trials & drug development due to the Inflation Reduction Act.

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Drug Target Review

JANUARY 8, 2024

We are at the forefront of drug development in an area of research called cellular rejuvenation, which is an approach that has the potential to address many diseases of ageing by restoring aged and injured cells to a more youthful and resilient state. Our lead program utilises OSK for the treatment of age-related optic neuropathies.

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Fierce BioTech

JANUARY 15, 2025

In this webinar, we will explore how high-multiplex spatial analysis tools are enabling Owkin to generate novel hypotheses and discoveries, and how the combination of spatial biology with AI could better inform a more optimal positioning of antibody-drug conjugates (ADCs) and respective combination treatments.

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Drug Target Review

SEPTEMBER 19, 2024

Could you give us an overview of IRLAB’s current drug development pipeline? It has a well-defined and strategically focused R&D pipeline of innovative treatments targeting various stages of Parkinson’s as they worsen over time. The fifth project is a potential new basic and first-line treatment that could replace levodopa.

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The Premier Consulting Blog

AUGUST 8, 2024

On July 31, 2024, the US Food and Drug Administration (FDA) announced Fiscal Year 2025 (FY2025) Prescription Drug User Fee Amendments of 2022 (PDUFA VII) fee rates for the review of human drug and biological product applications along with prescription drug program fees. i] United States Federal Register.

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Alta Sciences

APRIL 8, 2025

43 Advancing Vision Care With Formulation and Manufacture of Ocular Therapeutics pmjackson Tue, 04/08/2025 - 15:28 According to the WHO, at least a billion people are currently living with vision impairment from a preventable or treatable source. billion in 2021 to an estimated $68.93 billion by 2030.

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LifeSciVC

AUGUST 21, 2024

At this point of the year, the JPMorgan conference seems like ancient history and you are looking into flights for JPM 2025. Drug development is a long process, and patients are waiting. And importantly, breaks from treatment or treatment protocols can literally make or break trials. Was that only six months ago?

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