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To this end, the December 2024 draft guidance and a companion guidance published January 7, 2025, Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway (the January 2025 draft guidance ) describe FDAs latest thinking on what it means to conduct a confirmatory trial with due diligence.

FDA

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.

Disease

SugarCone Biotech

JUNE 19, 2025

Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). That is a decent hypothesis to explore and their early clinical data at ASCO 2025 looked interesting. Now to antibody-drug conjugates (ADC) where we saw some early programs with difficult toxicities. Stay tuned.

Therapies

Drug Target Review

JULY 8, 2025

As Vice President of Oncology Early Development (OED) at AbbVie , she leads the charge on translating bold scientific ideas into real-world impact – and she’s doing it with a focus on some of the most tenacious cancers out there. At that stage, the disease becomes more difficult to treat,” Eleni said.

Therapies

The Pharma Data

JUNE 18, 2025

Pushkal Garg to Lead Unified Research and Development Organization, Signaling Strategic Expansion into Next Phase of RNAi Therapeutics Innovation Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has announced the promotion of Pushkal Garg, M.D.,

RNA

Drug Target Review

JUNE 28, 2025

DARPin therapeutics were a prominent theme at AACR 2025 , with Molecular Partners presenting a trio of preclinical programmes spanning radiopharmaceuticals and immune cell engagers. ” With an IND filing and first-in-human trials expected in 2025, MP0712 is moving steadily towards clinical evaluation.

Science

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.

Disease

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.

Drugs

Drug Target Review

JULY 7, 2025

Non-human primates (NHPs), such as macaques and cynomolgus monkeys, have long served as a cornerstone in preclinical drug development due to their close genetic, anatomical and physiological resemblance to humans. Food and Drug Administration (FDA). National Primate Research Centers (NPRCs) Annual Report, 2021. Bailey J, et al.

Drug Research

The Pharma Data

JUNE 26, 2025

Located in Jiangsu Province, China, the Nantong site serves as a critical component of WuXi AppTec’s expansive Contract Research, Development, and Manufacturing Organization (CRDMO) platform. By integrating operational excellence with architectural brilliance, the site has become a blueprint for the future of industrial science campuses.

Laboratories

The Pharma Data

JUNE 9, 2025

Executive Commentary on Interim Results Dr. Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma, expressed optimism about the interim results. cm/year , which reflects a 4.23 cm/year increase from baseline. Source link

Trials

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. alone, more than 36,000 new diagnoses and approximately 12,000 deaths are anticipated in 2025.

FDA

Alta Sciences

JULY 7, 2025

ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs pmjackson Mon, 07/07/2025 - 21:31 New immunomodulatory drugs have the potential to cause serious immune-related adverse events, including cytokine release syndrome (CRS).

Immune Response

Alta Sciences

JULY 7, 2025

ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs pmjackson Mon, 07/07/2025 - 21:31 New immunomodulatory drugs have the potential to cause serious immune-related adverse events, including cytokine release syndrome (CRS).

Immune Response

LifeSciVC

JUNE 12, 2025

2025 holds to be a transformative year for us, with the potential for multiple milestones to come to fruition, with the biggest value inflection being the interim clinical data from our lead asset KRRO-110, a potential best in class compound for patients with Alpha-1 antitrypsin deficiency (AATD). Over the last 4.5

RNA

New Drug Approvals

AUGUST 7, 2025

5] Repotrectinib was approved for medical use in the United States in November 2023, [5] [6] and in the European Union in January 2025. [3] These mutations restrict the ability of drugs to bind to their targets, ultimately resulting in the advancement of tumors. Retrieved 23 May 2025. 14 January 2025. in DMF (4.0

FDA

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.

Drug Development

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. Ibtrozi has also shown the ability to help people whose cancer has spread to the brain, a leading cause of disease progression, the company said.

FDA

FDA Law Blog: Drug Discovery

FEBRUARY 11, 2025

However, as we suggested in a recent blog post , the statute did not actually provide any limitations on FDAs authority to grant rare pediatric disease designations , only its authority to grant vouchers. In the meantime, the Pink Sheet confirmed that FDA was continuing to review rare pediatric disease designation requests.

Disease

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. Now, Vor is reestablishing itself as an autoimmune disease company.

Disease

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. Kennedy Jr., Kennedy Jr. Kennedy Jr.

Vaccine

BioPharma Drive: Drug Pricing

JULY 18, 2025

Published July 18, 2025 Delilah Alvarado Staff Reporter post share post print email license Food and Drug Administration Commissioner Martin Makary speaks on stage during The Semafor 2025 World Economy Summit on April 24, 2025 in Washington, DC. By Jacob Bell • July 1, 2025 Keep up with the story. inhibitors.

Therapies

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. The disease, which affects an estimated 10,000 to 20,000 people in the U.S.,

Drugs

BioPharma Drive: Drug Pricing

JULY 30, 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 News roundup Madrigal buys into GLP-1 from CSPC; Apellis wins expanded drug approval The Hong Kong-based CSPC will receive $120 million from Madrigal under the deal.

Drugs

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.

Clinical Trials

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.

Clinical Trials

BioPharma Drive: Drug Pricing

JUNE 26, 2025

Published June 26, 2025 Ned Pagliarulo Lead Editor post share post print email license An Incyte researcher works in a laboratory. On June 26, 2025, the company announced the replacement of longtime CEO Hervé Hoppenot with Bill Meury. Last year, Jakafi brought in nearly $2.8 billion in sales. Track funding rounds here.

Therapies

Alta Sciences

JUNE 26, 2025

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Pharmacokinetics

The Pharma Data

JUNE 6, 2025

Drivers of Market Growth: From Precision Medicine to Cost Optimization The shift toward targeted therapies , rare diseases , and personalized medicine is fundamentally altering pharmaceutical commercialization strategies. CNS Disorders and Autoimmune Diseases : Where nuanced communication and long-term patient management are key.

Pharmaceutical Companies

Alta Sciences

JUNE 26, 2025

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Pharmacokinetics

Conversations in Drug Development Trends

APRIL 29, 2025

On the last day of February every year is Rare Disease Day , a dedicated day to celebrate the rare disease community, including patients, families, caregivers, clinicians, researchers, regulators, and more. The goal is to build community, share ideas and resources, and advance research and innovation.

Disease

Codon

JULY 14, 2025

And some of the remedies are still practiced today: the treatment for Guinea-worm disease was, and still is , to wrap the emerging end of the worm around a stick and slowly pull it out. Drugs developed in Eichengrün’s laboratory were tested at Bayer’s pharmacological laboratory, led by Heinrich Dreser.

Treatment

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

The Pharma Data

AUGUST 1, 2025

The repurchase, announced just days after the company’s board of directors authorized the new buyback program on July 30, 2025, marks one of the most significant share repurchase commitments by a biotech company this year. On August 4, 2025, United Therapeutics will make a total upfront payment of $1 billion to Citi.

Government

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.

Treatment

BioPharma Drive: Drug Pricing

AUGUST 11, 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 Sponsored Regulatory uncertainty and access challenges changing go-to-market models, DTC, and DTP Published Aug. By BioPharma Dive staff • Updated July 2, 2025 Keep up with the story.

Marketing

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. As a result, regulatory assessment is ongoing and the FDA granted orphan drug designation in March 2025.

Trials