New Drug Approvals

APRIL 25, 2025

3] Inavolisib was approved for medical use in the United States in October 2024. [3] The difluoromethyl group can interact with the hydroxyl group presented on Ser774 (conserved) in p110, which is 3.2 3] [6] [20] [21] The drug application was granted priority review and breakthrough therapy designations by the FDA. [3]

FDA

New Drug Approvals

JUNE 11, 2025

The FDA rejected approval for gepirone in 2002 and 2004. [5] 5] It was submitted for the preregistration (NDA) phase again in May 2007 after adding additional information from clinical trials as the FDA required in 2009. 12] However, in March 2016, the FDA reversed its decision and gave gepirone ER a positive review. [13]

FDA

The Pharma Data

JUNE 15, 2025

The new results were presented in a late-breaking oral presentation (Abstract #LB4001) at the 2025 European Hematology Association (EHA) Congress. 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.

Therapies

The Premier Consulting Blog

JANUARY 14, 2025

These resources provide accurate and current strategies that strongly represent FDAs standpoint and have routinely been leveraged by Premiers team of strategists when guiding sponsors through their development plans. The summary presented in the table below features some of these initiatives. Program What is it?

Clinical Trials

New Drug Approvals

APRIL 18, 2025

2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2] 1 December 2024.

FDA

Codon

NOVEMBER 3, 2024

Still, while these developments provide cause for hope, each drug or vaccine faces a variety of challenges, ranging from financial incentives for clinical trials to the difficulty of discovering new antimicrobials that pass safety and efficacy tests. A phase 3 clinical trial for M72/AS01E, funded by the Gates Foundation, began this year.

Vaccine

New Drug Approvals

JULY 8, 2025

Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. Clinical Trials: Pritelivir mesylate is currently under extensive study to evaluate its efficacy and safety profile, with promising results in early clinical trials.

Virus

The Pharma Data

JUNE 13, 2025

FDA Approves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S.

FDA Approval

New Drug Approvals

APRIL 19, 2025

Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 Treatment of phenol 143 with bromo epoxide 144 in the present of K2CO3 afforded ether 145 in 76% yield. Jump up to: a b “Novel Drug Approvals for 2024” U.S. AOP Health.

FDA

Science Daily: Pharmacology News

JUNE 21, 2025

Fresh concerns have emerged about the platelet studies underpinning the FDA approval of ticagrelor, AstraZeneca's multibillion-dollar heart drug. A new BMJ investigation reveals data discrepancies, missing lab readings, and questions about the integrity of the trial process.

FDA

PLOS: DNA Science

MARCH 28, 2024

The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis. Results from the study that led to the FDA approval appeared in The Lancet Neurology in April 2024 with commentary.

FDA Approval

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

Clinical Trials

The Pharma Data

MAY 22, 2023

Teva to Present Data Demonstrating UZEDY™ (risperidone) Extended-Release Injectable Suspension Significantly Prolonged Time to Impending Relapse for Adults with Schizophrenia at the American Psychiatric Association’s 2023 Annual Meeting Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. once-monthly dosing) and 2.7

FDA Approval

The Pharma Data

OCTOBER 29, 2021

Novartis blazoned moment that the US Food and Drug Administration (FDA) approved Scemblix ® (asciminib) for the treatment of habitual myeloid leukemia (CML) in two distinct suggestions. In cases with Ph CML-CP who had endured resistance or dogmatism to at least two TKIs, the ASCEMBL trial showed that1-3.Scemblix

FDA Approval

The Pharma Data

NOVEMBER 27, 2020

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. The FDA granted approval under the accelerated approval regulation. NEW YORK, Nov. Contraindications.

FDA Approval

The Pharma Data

JULY 8, 2021

The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language). Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Clinical Trials

The Pharma Data

MARCH 11, 2021

Eli Lilly and Company (NYSE: LLY) today announced that data from programs across its oncology portfolio and pipeline will be presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, to be held virtually April 10-15, 2021. LY3484356, an oral SERD, is currently being studied in a Phase 1/2 clinical trial.

Research

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies

The Pharma Data

AUGUST 7, 2021

Nexviazyme has been shown in clinical trials to provide patients with improvements in respiratory function and walking distance. LOPD symptoms may present at any age. A key secondary endpoint in the trial measured functional endurance with the 6-minute walk test (6MWT). Nexviazyme demonstrated improvements in pivotal study.

FDA Approval

Drug Target Review

JANUARY 8, 2024

We hope to initiate the first human clinical trials with our lead indications in 2025, which would represent a monumental step forward for the field of cellular rejuvenation. What’s next for Life Bioscience’s preclinical studies? Most recently, Jerry was President and CEO for Neos Therapeutics, Inc.,

Science

The Pharma Data

JANUARY 7, 2021

Hosts a Panel Discussion on Clinical Trials with Neil Solomons, M.D., The Company is currently seeking FDA approval of voclosporin for the potential treatment of lupus nephritis (LN). Wainwright Virtual BioConnect 2021 Conference. On-demand fireside chat available beginning Monday, January 11, 2021 at 6:00 a.m. EST; and.

Pharmaceuticals

The Pharma Data

SEPTEMBER 8, 2021

–Results from a post-hoc analysis of the Phase 3 PREEMPT trials evaluate the use of BOTOX® (onabotulinumtoxinA) for chronic migraine –These data further demonstrate AbbVie’s commitment to harnessing and sharing innovative science and working to advance treatment options for people with migraine across the migraine spectrum.

Doctors

The Pharma Data

SEPTEMBER 2, 2020

Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations. Other data to be presented will show high treatment persistence and strong adherence for patients treated with OCREVUS compared to other DMTs in real-world settings.

Clinical Trials

Vial

JUNE 12, 2024

PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. Food and Drug Administration (FDA) increasingly look to patients to understand how they describe their health status. What are PROs in clinical trials?

Clinical Trials

The Pharma Data

OCTOBER 15, 2020

The findings were presented at TCT Connect , the 32nd annual scientific symposium of the Cardiovascular Research Foundation. In the first study, presented by Hemindermeet Singh, MD, of Ascension St. Since FDA PMA approval, Abiomed (NASDAQ: ABMD) has collected data on nearly 100% of U.S. View the full release here: [link].

Hospitals

The Pharma Data

SEPTEMBER 8, 2020

Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Roche’s Chief Medical Officer and Head of Global Product Development. “In

Clinical Trials

The Pharma Data

AUGUST 30, 2021

Results reinforce well-established safety profile of Dupixent – the first ever biologic medicine for atopic dermatitis currently approved for patients as young 6 years old. The trial demonstrated similar safety results to the known safety profile of Dupixent in atopic dermatitis. Yancopoulos, M.D., In 2016, the U.S.

Trials

Pharmaceutical Development Group

DECEMBER 19, 2021

Effective use of Real World Data (RWD) and Real World Evidence (RWE) can reduce costs of Pharmaceutical Drug and Biologic Products and Process Development, Drug Development Services , expedite a FDA Pre-Submission Review, and lead to FDA Approval. Author Information William E. Spanogle, Ph.D. Spanogle, Ph.D.

FDA Approval

The Pharma Data

JANUARY 13, 2021

Fast Track designation is well-timed, as we anticipate starting our Phase 2 clinical trial in hospitalized COVID-19 patients this month, and should help bring Brilacidin to patients faster in these dire times.”. Brilacidin, a versatile compound with broad therapeutic potential, is in a new chemical class called defensin-mimetics.

Clinical Trials

The Pharma Data

FEBRUARY 18, 2022

These data (Presentation #DOP44) will be presented at the 17 th Congress of the European Crohn’s and Colitis Organisation (ECCO), taking place February 16-19, 2022. Additional Bristol Myers Squibb-sponsored abstracts presented at the ECCO 2022 Congress can be accessed online here. About Bristol Myers Squibb.

Clinical Trials

The Pharma Data

OCTOBER 27, 2021

Kymriah was preliminarily granted orphan medicinal product designation by the European Commission (EC) forFL.However, Kymriah would have the occasion to present an important treatment option for those cases with r/ r FL in need of potentially definitive issues, If approved in this implicit third suggestion.

FDA

The Pharma Data

APRIL 28, 2023

Teva and MedinCell Announce FDA Approval of UZEDY™ (risperidone) Extended-Release Injectable Suspension, a Long-Acting Subcutaneous Atypical Antipsychotic Injection, for the Treatment of Schizophrenia in Adults Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. UZEDY will be available in the U.S.

FDA Approval

The Pharma Data

APRIL 7, 2021

today announced new data from its expansive neuroscience portfolio will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually from April 17-22. Key AbbVie abstracts and presentation details for the 2021 AAN Annual Meeting program are outlined below. Presentation Details. All times CT.

Clinical Trials

The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.

Disease

The Pharma Data

JANUARY 13, 2021

I am pleased with the clinical progress we are making with this program and we are planning to present interim data from the ongoing Phase 1/2 dose escalation trial later this year.”. About the Phase 1/2 Trial for HPN217. Dose escalation for HPN217 in the Phase 1/2 clinical trial is progressing rapidly.

Treatment

The Pharma Data

APRIL 8, 2021

Additional presentations on investigational programmes, including Alzheimer’s disease and Huntington’s disease, help advance scientific understanding of neurological disorders. Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA.

Clinical Trials

The Pharma Data

JUNE 7, 2022

The new findings from the Phase 3 clinical trials (ADvocate 1 and 2) showed eight out of ten patients who achieved clinical response (EASI-75*) with lebrikizumab monotherapy at 16 weeks maintained skin clearance at one year of treatment with the once every two weeks or four weeks regimen. Source link: [link].

Trials