Drug Target Review

JULY 1, 2025

These digital twins are created for each trial participant using their baseline data – regardless of whether they are assigned to the placebo or treatment arm – and simulate how that individual would have responded under control conditions. Integration is straightforward,” Herne notes.

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Chemical Biology and Drug Design

APRIL 4, 2025

Currently, the FDA has granted approval for the use of the small molecule inhibitor Ivosidenib (AG-120) in the treatment of IDH1-mutated AML and cholangiocarcinoma. Although AG-120 has benefited patients clinically, drug resistance has gradually emerged and has become a major problem in the treatment of mutant IDH1 (mIDH1) diseases.

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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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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.

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Drug Target Review

APRIL 7, 2025

Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.

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Drugs.com

MAY 27, 2025

percent for the treatment of plaque psoriasis of the scalp and body in adult and pediatric patients 12 years of age and older.Through. TUESDAY, May 27, 2025 -- The U.S. Food and Drug Administration has approved Zoryve (roflumilast) topical foam 0.3

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Drug Target Review

NOVEMBER 25, 2024

As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. This is exciting because often resistance builds up to these other treatments.

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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).

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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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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. FDA news release, “FDA announces plan to phase out animal testing requirement for monoclonal antibodies and other drugs.”

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New Drug Approvals

JUNE 24, 2025

Safety: Diarrhea was the most frequent adverse event, but serious adverse events were balanced across treatment groups. FDA Designation: Nerandomilast received Breakthrough Therapy Designation from the FDA for the treatment of IPF. Nerandomilast CAS 1423719-30-5 C 20 H 25 ClN 6 O 2 S Molecular Weight 448.97 United States.

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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). a global oncology-focused biopharmaceutical company, has received a significant regulatory milestone from the U.S.

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FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. FDA did not object to the timing of the respective submissions in either letter. What Happened?

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The Premier Consulting Blog

NOVEMBER 6, 2024

Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. What regulatory division governs ICPs? ICPs are subject to rigorous regulatory scrutiny due to their complexity.

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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

Sunvozertinib , sold under the brand name 舒沃哲, among others is an anti-cancer medication used for the treatment of non-small-cell lung cancer. [2] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] Food and Drug Administration (FDA).

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The Pharma Data

JUNE 25, 2025

Designed using the same fundamental mechanism as Biogen’s approved SMA treatment SPINRAZA (nusinersen), salanersen represents a next-generation approach aimed at greater potency and less frequent dosing—potentially requiring only once-yearly administration. Treatment-naïve individuals, to evaluate efficacy as a first-line therapy.

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The Pharma Data

JUNE 12, 2025

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

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The Pharma Data

JUNE 9, 2025

A New Treatment Option for a Debilitating Joint Tumor TGCT is a rare, debilitating condition that primarily affects young and middle-aged adults, often striking individuals in their prime working years. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

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New Drug Approvals

MAY 17, 2025

Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] 4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [7] Food and Drug Administration (FDA). Food and Drug Administration (FDA) (Report). 25 April 2023.

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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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New Drug Approvals

JUNE 11, 2025

4] It was approved for the treatment of major depressive disorder in the United States in September 2023. [4] 4] This came after the drug had been rejected by the Food and Drug Administration (FDA) three times over two decades due to insufficient evidence of effectiveness. [5] The FDA rejected approval for gepirone in 2002 and 2004. [5]

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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).

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Broad Institute

JULY 21, 2025

There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& The team used a scalable approach to develop prime editing treatments that directly repaired five different AHC-causing genetic mutations. The results really exceeded our expectations,” said Sakai. “It

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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. It is characterized by early-onset, treatment-resistant seizures, beginning as early as six weeks of age, and profound global neurodevelopmental impairment.

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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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BioPharma Drive: Drug Pricing

JULY 8, 2025

Kymera, which hopes to make oral drugs with biologic-like efficacy, released data for KT-621 in June , which showed treatment was well-tolerated in healthy volunteers. Both are developing treatments that draw on the idea of antibody-drug conjugates, but swap out the chemotherapy toxin for a protein degrader.

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The Pharma Data

JUNE 6, 2025

This outcome suggests sibeprenlimab’s potential to not only delay disease advancement but also improve long-term renal outcomes, addressing a critical unmet need for patients with IgAN, many of whom currently face limited treatment options. Treatment-emergent adverse events (TEAEs) occurred in 76.3% in the placebo group.

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Metabolite Tales Blog

JUNE 6, 2025

Iptacopan is a complement B factor inhibitor approved in the US and EU for the treatment of paroxysmal nocturnal hemoglobinuria and proteinuria. In March 2025, oral Fabhalta was approved by the FDA for treatment of C3 glomerulopathy, making it the first and only drug approved for this condition.

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SugarCone Biotech

JUNE 19, 2025

Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. Toxicities were challenging enough to cause 13% of patients to reduce dose or skip doses and 3% to discontinue treatment. months (versus 20% ORR and 8.3 Stay tuned.

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The Pharma Data

JUNE 18, 2025

By combining Neoclease’s AI-designed gene-editing platform with our technologies, regulatory expertise, and process support, we aim to help accelerate new treatments that minimize side effects and maximize effectiveness for patients.”

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Alta Sciences

MARCH 4, 2025

This was in 2006, at a time when the FDA guidances on these topics had not yet been published. Over time, this group of professionals evolved and grew, having regular stakeholder interactions with the FDA and Controlled Substances staff to discuss requirements for drug developers. corticosteroids, beta-blockers, antidepressants).

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New Drug Approvals

APRIL 25, 2025

8] Therefore, it may serve as a new addition to combination therapy with conventional cancer treatment, such as chemotherapy. Combining inavolisib with palbociclib and fulvestrant might improve treatment of breast cancer. [14] Food and Drug Administration (FDA). This results in a very high selectivity regarding PI3K isoforms. [4]

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Broad Institute

JUNE 24, 2025

Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. Some of these treatments, like K.J.’s, The team that treated K.J. Today, K.J.

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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. Food and Drug Administration (FDA) as a monotherapy for children with achondroplasia.

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New Drug Approvals

MAY 9, 2025

Randomization was stratified based on whether the ESR1 mutation was detected or not, prior treatment with fulvestrant, and presence of visceral metastasis. [2] 2] The FDA granted the application for elacestrant priority review and fast track designations. [2] Food and Drug Administration (FDA). 1] [4] It is taken by mouth. [1]

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The Pharma Data

JUNE 24, 2025

Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers. These standards are widely recognized by regulatory agencies such as the FDA and EMA for use in analytical method development, validation, and quality control.

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