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. The Natural History Studies Grants Program funds natural history studies that collect gather data on how rare diseases progress over time without treatment.

Clinical Development

Chemical Biology and Drug Design

JANUARY 30, 2025

The potential therapeutic efficacy of luteolin in the treatment of cervical cancer has been identified. Therefore, we aim to elucidate the mechanism of action of luteolin in the treatment of cervical cancer through a comprehensive approach that integrates metabolomics with bioinformatics.

Bioinformatics

Crown Bioscience

JULY 17, 2025

Oncology drug approvals in H1 2025 In the first half of 2025, the FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 16 novel drugs , with half of these drugs related to the treatment of cancer.

FDA Approval

BioPharma Drive: Drug Pricing

JUNE 30, 2025

The acquisition will expand AbbVie's exploration of in vivo CAR-T therapy, an ambitious approach that could sidestep some of the difficulties presented by current cell treatments.

Therapies

Drug Target Review

JULY 21, 2025

Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. This makes it difficult to identify relevant and effective treatments.

Drug Development

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).

Therapies

Drug Target Review

DECEMBER 23, 2024

While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. Properly managing these toxicities is crucial to enhancing the safety and therapeutic effectiveness of ADC treatments.

Research

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.

FDA Approval

Drug Target Review

JUNE 6, 2025

At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. The founding of RadioMedix was born from a recognition of unmet needs in cancer treatment.

Therapies

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.

Clinical Development

Drug Target Review

APRIL 30, 2025

In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. Kosten Digital was established to bridge this gap.

Drugs

The Pharma Data

JUNE 13, 2025

Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies.

Therapies

Drug Target Review

JUNE 16, 2025

Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.

Disease

Drug Target Review

DECEMBER 20, 2024

These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes. In 2025, I think well see breakthroughs that allow us to make significant advances in rare diseases, where the data is small.

Clinical Trials

BioPharma Drive: Drug Pricing

JUNE 17, 2025

The deal centers around a medicine that could “shift the treatment paradigm for cardiovascular disease,” Lilly said, but also follows a yearslong in decline in Verve’s share price amid skepticism over the need for such a therapy.

Disease

Broad Institute

JUNE 11, 2025

Broad Institute and Bayer extend their cardiovascular alliance By Leah Eisenstadt June 11, 2025 Breadcrumb Home Broad Institute and Bayer extend their cardiovascular alliance Partnership will continue with renewed focus on developing new cardiovascular therapies.

Disease

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. Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers.

Therapies

Drug Target Review

MARCH 4, 2025

Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach.

Therapies

Drug Target Review

JULY 8, 2025

Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up. mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As

Therapies

Drug Target Review

OCTOBER 30, 2024

8 With the widespread use of induced pluripotent stem cells (iPSCs), organoids can now be generated from the skin or blood cells of both healthy volunteers and disease patients, opening the door to studying various diseases and creating individually customised potential treatments. Organoids in cancer research. Nat Rev Cancer.

Treatment

New Drug Approvals

JULY 5, 2025

This means it can block estrogen receptor activity and also degrade the receptor itself, potentially offering a more effective treatment approach. Clinical Trials: Palazestrant is currently in clinical trials, including Phase 1/2 and Phase 3 studies, for the treatment of ER+, HER2- metastatic breast cancer. ribociclib).

Small Molecule

Broad Institute

MAY 21, 2025

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. Adapted from an Allen Institute press release.

Disease

Crown Bioscience

JUNE 23, 2025

Drug resistance remains one of the most pressing challenges in oncology drug development, as it is the leading cause of treatment failure. This article explores the role in vitro models are playing in the fight against drug resistance and explains how they can accelerate the development of successful novel therapies.

Drugs

Drug Target Review

JULY 24, 2025

Antibody therapies have transformed cancer treatment, yet their limits are becoming increasingly clear – particularly when tumours evolve, evade immune detection or develop resistance to existing drugs. The presence of p95HER2 – a shortened version of the receptor that still drives tumour growth – makes treatment even more difficult.

Immune Response

Chemical Biology and Drug Design

MAY 10, 2025

Although it is currently only used in veterinary therapy, carprofen proves to be a molecule with a versatile therapeutic potential for human pharmacotherapy. With ongoing research pushing the boundaries of its potential, carprofen remains a promising candidate for innovation in drug development and treatment strategies.

Small Molecule

Chemical Biology and Drug Design

JUNE 17, 2025

Flavonoids exhibit significant potential in cancer therapy by inhibiting MMP-2 and MMP-9 activity and expression, primarily through interactions with their active sites and modulation of the MAPK signaling pathway. ABSTRACT Last few decades, extensive research efforts have been dedicated to uncovering novel cancer treatments.

Regulations

The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. According to Merck, the addition of WINREVAIR to background therapy resulted in a meaningful improvement in patient outcomes, particularly in delaying disease progression and reducing the risk of severe clinical events.

Trials

Drug Target Review

JULY 3, 2025

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

Drug Development

The Pharma Data

JUNE 16, 2025

Teva and Fosun Pharma Forge Strategic Partnership to Develop and Commercialize Innovative Anti-PD1-IL2 Therapy (TEV-56278) in Immuno-Oncology Teva Pharmaceutical Industries Ltd. TEV-56278 is a first-in-class anti-PD1-IL2 ATTENUKINE therapy designed by Teva’s internal team of innovative scientists.

Therapies

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

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

The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

Treatment

Drug Target Review

DECEMBER 6, 2024

In cases where control or treatment groups show large variability, this could reflect true biological responses to a treatment, and removing such data could reduce the accuracy of the analysis. It is crucial during this phase to strike a balance between removing erroneous data and preserving meaningful biological variability.

RNA

Covalent Modifiers

JANUARY 9, 2025

Drugging such undruggable targets is essential to develop new therapies for diseases where current treatment options are limited or nonexistent. Tomonori Tamura, Masaharu Kawano, and Itaru Hamachi Chemical Reviews 2024 DOI: 10.1021/acs.chemrev.4c00745

Drugs

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. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis.

Therapies

Crown Bioscience

JULY 28, 2025

Antibody-drug conjugates (ADCs) are among the most exciting advances in target cancer therapy. They combine the specificity of monoclonal antibodies with the cytotoxic potency of small molecule drugs to focus treatment on malignant cells while reducing damage to healthy cells.

Small Molecule

The Pharma Data

JUNE 18, 2025

The investigational ADC is built using Daiichi Sankyo’s proprietary DXd technology and represents one of the newest generation of targeted cancer therapies. Current treatment strategies for mCRPC typically begin with ARPIs such as enzalutamide or abiraterone, followed by taxane-based chemotherapy , most commonly docetaxel.

Trials