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.,

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SCIENMAG: Medicine & Health

JUNE 15, 2023

A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options. Credit: Vanderbilt University Medical Center A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options.

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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.

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ASPET

SEPTEMBER 28, 2023

Status epilepticus (SE) is a life-threatening development of self-sustaining seizures that becomes resistant to benzodiazepines when treatment is delayed. Benzodiazepine pharmacoresistance is thought in part to result from internalization of synaptic GABA A receptors, which are the main target of the drug.

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New Drug Approvals

APRIL 11, 2025

2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] 2] [3] Names Fitusiran is the international nonproprietary name. [4] Fitusiran 1711.0g/mol,

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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. For many years, systemic corticosteroids have been the mainstay of BP treatment.

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The Pharma Data

JUNE 27, 2025

mg/mL solution for injection) across the European Union (EU), allowing for extended treatment intervals of up to six months for individuals diagnosed with neovascular (wet) age-related macular degeneration (nAMD) and visual impairment due to diabetic macular edema (DME).

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DrugBank

JUNE 13, 2024

These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects.   Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.

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Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.

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ASPET

NOVEMBER 17, 2023

Benzodiazepine pharmacoresistance develops when treatment of status epilepticus (SE) is delayed. This response may result from gamma-aminobutyric acid A receptors (GABA A R) internalization that follows prolonged SE; this receptor trafficking results in fewer GABA A R in the synapse to restore inhibition.

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Drug Discovery Today

NOVEMBER 10, 2022

A team of surgeons and scientists from the UK, Sweden and Canada, funded by Rinri Therapeutics, has confirmed secure surgical access to the central core of the human cochlea The research, published in Scientific Reports, is critical to the first in-human trials of new cell, gene and drug therapies for the inner ear, and will assist with treatment for (..)

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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.

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The Pharma Data

JULY 3, 2025

The results, recently published in the International Journal of Molecular Sciences , come from a collaborative effort between researchers at Erlangen University Hospital and Berlin-based biotech firm AudioCure. Importantly, the treatment also appeared to reverse the underlying physiological damage believed to contribute to tinnitus.

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New Drug Approvals

JUNE 29, 2025

The resulting mixture was stirred at an internal temperature of about 65° C. was further added thereto, and then the resulting mixture was stirred at an internal temperature of 79° C. was added dropwise at an internal temperature of 22° C. The resulting mixture was stirred at an internal temperature of 19° C. for 4 hours.

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The Premier Consulting Blog

JANUARY 14, 2025

As the oncology landscape continues to evolve, the OCE aims to improve access to safe, effective, and cutting-edge therapies through streamlined regulatory processes and patient-centered approaches. These include Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation, and the Accelerated Approval Pathway (AAP).

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The Pharma Data

JUNE 22, 2025

The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. With new non-factor therapeutic options, many patients may feel uncertain about switching treatments.

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The Pharma Data

JUNE 22, 2025

The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., Importantly, the therapy demonstrated a favorable safety profile, with no thromboembolic events reported so far. Engineered by Chugai Pharmaceutical Co.,

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Drug Patent Watch

DECEMBER 9, 2024

Taxol: From Pacific Yew to Cancer Treatment Taxol (paclitaxel), a widely used cancer drug, was originally isolated from the bark of the Pacific yew tree. Artemisinin: Nobel Prize-Winning Malaria Treatment Artemisinin, derived from the sweet wormwood plant, has become a crucial treatment for malaria.

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments.

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The Pharma Data

JUNE 27, 2025

The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. These data offer new hope to the CDD community, which has long struggled with a lack of effective seizure control therapies.

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The Pharma Data

JUNE 6, 2025

Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., from June 21 to 25, 2025.

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The Pharma Data

JUNE 16, 2025

a principal investigator for the TRANSCEND FL study and a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center in New York. This high durability highlights the sustained Benefit provided by liso-cel in a population typically faced with poor prognosis after multiple lines of standard therapy. months, and 24.5

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The Pharma Data

JUNE 18, 2025

The double-blind treatment period lasted 24 weeks, during which both efficacy and adverse events (AEs) were closely monitored. Primary Endpoint Met: Improved Tolerability with Atogepant At the core of the TEMPLE study was the investigation of treatment discontinuation rates due to adverse events, a key measure of real-world drug tolerability.

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PPD

DECEMBER 16, 2024

Rising costs have become a persistent challenge for drug developers, driven by a combination of internal and external pressures that have intensified in recent years. Each delay not only increases costs but also jeopardizes a treatments viability, underscoring the urgency for efficient timelines and streamlined processes.

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SCIENMAG: Medicine & Health

JUNE 21, 2023

New research published in the Journal of Internal Medicine demonstrates that optogenetics—which uses light-sensitive proteins to control the activity of targeted cells—is a promising shock-free approach to treating atrial fibrillation (AF), or an irregular, often rapid heart rate, for immediate restoration of regular rhythm.

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The Pharma Data

JUNE 18, 2025

This leadership transition arrives at a pivotal moment in Alnylam’s evolution, as the company builds upon the recent commercial success of AMVUTTRA® (vutrisiran) —a landmark treatment for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). He holds a B.A.

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The Pharma Data

JUNE 24, 2025

The strategic funding arrangement is designed to support the continued advancement and global commercialization of Revolution Medicines’ RAS(ON) inhibitor portfolio, including its lead candidate daraxonrasib, as the company scales its operations independently across the international oncology market.

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Drug Target Review

DECEMBER 11, 2024

By employing advanced analytical techniques like high-resolution mass spectrometry (HRMS) and carefully calibrating systems with optimal internal standards (IS), we effectively mitigate matrix effects and provide reliable preclinical toxicology data. These solutions must contain analytes and internal standards at known concentrations.

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Conversations in Drug Development Trends

OCTOBER 29, 2024

In this way, regulators can evaluate proposed trial designs and therapies in terms of what value the trial will provide to participants and how well new treatments might serve the affected populations.

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The Pharma Data

JUNE 15, 2025

According to the data, the combination demonstrated a high ORR and a strong depth of response in a patient population with limited treatment options and poor outcomes under standard care. were penta-drug refractory, having been exposed to additional lines of treatment. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.

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New Drug Approvals

APRIL 2, 2025

2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment. The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA. Suzetrigine is taken by mouth. [1]

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Common Sense for Drug Policy Blog

APRIL 21, 2024

Agonist Treatment for Cannabis Use Disorder "Cannabinoid agonist treatment is unlikely to be an approach relevant to all cannabis users seeking treatment, as evidenced by the large numbers of individuals who did not complete the study screening process, and the modest 12-week treatment retention rates. JAMA Intern Med.

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Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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The Pharma Data

JUNE 11, 2025

This decision represents a pivotal moment in the evolution of treatment convenience for patients with certain B-cell malignancies, while reinforcing BRUKINSA’s stronghold in the BTK inhibitor market. Notably, BRUKINSA has emerged as the market leader in new CLL patient starts across all lines of therapy in the U.S. for the first time.

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The Pharma Data

JUNE 27, 2025

The alert follows a detailed safety review and recommendation from the European Medicines Agency (EMA) , which concluded that non-arteritic anterior ischemic optic neuropathy (NAION) —a rare but serious eye condition resulting in permanent vision loss—should now be officially recognized as a very rare side effect of semaglutide therapy.

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Elrig

MARCH 25, 2025

GOLD Lab collaborates with leading international consortia, including Genomics England and FANTOM (Functional Annotation of the Mammalian Genome). His team has developed CRISPR-Cas and cancer-driver methods for the discovery of oncogenic lncRNAs that are promising targets for therapeutic oligonucleotides.

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The Pharma Data

JUNE 27, 2025

Biogen Unveils New Data Underscoring the Long-Term Benefits and Advancements of Nusinersen in Spinal Muscular Atrophy Treatment Biogen , a global leader in neuroscience, has announced compelling new data that reinforces the clinical value and therapeutic potential of nusinersen, a pioneering treatment for spinal muscular atrophy (SMA).

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