DrugBank

JUNE 18, 2025

Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. While international agencies strive for harmonization through organizations such as the International Council for Harmonisation (ICH), regulatory frameworks differ significantly across regions.

Drug Development

New Drug Approvals

JULY 25, 2025

Corticosteroid therapy is the current standard of care for DMD despite relatively high rates of adverse effects. 75,76 This also results in decreased glucocorticoid receptor-drive transactivation, ultimately improving the safety profile of vamorolone as compared to other corticosteroid therapies. 77 Readily available steroid 9.1

FDA

Drug Target Review

MARCH 17, 2025

However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. The drug’s pharmacokinetics (PK) and pharmacodynamics (PD) are closely linked.

Treatment

New Drug Approvals

APRIL 18, 2025

Compound 8A was previously described in International Publication Number WO2010/125414 by Sanofi-Aventis.Example 1: Preparation of 4-(2-chloro-4-methoxy-5-methylphenyl)-N-[(1S)-2-cyclopropyl-1-(3-fluoro-4-methylphenyl)ethyl]-5-methyl-N-prop-2-ynyl-1,3-thiazol-2-amine (Compound 1), See FIG. New Drug Therapy Approvals 2024 (PDF).

FDA

New Drug Approvals

APRIL 19, 2025

“Pharmacokinetics of landiolol hydrochloride, a new ultra-short-acting beta-blocker, in patients with cardiac arrhythmias” Clinical Pharmacology and Therapeutics. New Drug Therapy Approvals 2024 (PDF). “Metoprolol Therapy and CYP2D6 Genotype” In Pratt VM, McLeod HL, Rubinstein WS, et al. 32 (2): 828.

FDA

The Pharma Data

JUNE 18, 2025

Omaveloxolone, already marketed as SKYCLARYS® , is the only approved therapy for Friedreich ataxia in adults and adolescents 16 years and older in more than 40 countries, including the United States and members of the European Union. Head of the Neuromuscular Development Unit at Biogen. Source link

Trials

The Pharma Data

NOVEMBER 17, 2020

The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies.

Pharmacokinetics

LifeSciVC

APRIL 24, 2024

Suggested resources for this first pass genetics diligence include OMIM, GWAS Catalog, DisGeNET, OpenTargets, Genebass, and the International Mouse Phenotyping Consortium. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., in liver, in CNS)? with antibodies), or correction (e.g.,

Production

New Drug Approvals

DECEMBER 24, 2023

1] Society and culture Names Eplontersen is the international nonproprietary name. [9] “Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen” Neurology and Therapy. 12 (1): 267–287. doi : 10.1007/s40120-022-00414-z.

Clinical Trials

The Pharma Data

AUGUST 9, 2021

Since 40% of people with DLBCL relapse after initial therapy, achieving meaningful treatment effects in the front-line setting has the potential to be transformative,” said Levi Garraway, M.D., Safety outcomes were consistent with those seen in previous trials. Roche’s Chief Medical Officer and Head of Global Product Development.

International

The Pharma Data

JULY 12, 2021

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinical development program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. No Pharmacokinetic Interaction Between Novel NNRTI MK-8507 and Islatravir.

Pharmacokinetics

Advarra

JUNE 20, 2023

cell and gene therapies), with other therapeutic areas then pushing it further. Because of this, oncology researchers may only have access to minimal existing scientific literature related to cell-based data and animal testing to predict an investigational therapy’s effect on human factors.

Trials

The Pharma Data

MARCH 13, 2021

Takeda Pharmaceutical Company Limited ( TSE:4502/NYSE:TAK ) (“Takeda”) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

Treatment

New Drug Approvals

OCTOBER 24, 2023

1] [9] [10] Names Etrasimod is the international nonproprietary name. [11] “Etrasimod as induction and maintenance therapy for ulcerative colitis (ELEVATE): two randomised, double-blind, placebo-controlled, phase 3 studies” Lancet. 11] SYN ACS Med. twitter +919321316780 call whatsaapp EMAIL. 401 (10383): 1132–1133.

FDA

Alta Sciences

SEPTEMBER 13, 2023

COMMONLY USED PAIN MODELS As pain models continue to expand, there are still many tried and tested ways to achieve the most accurate results possible, such as pain stimulation, pharmacodynamics (PD), and pharmacokinetics (PK) for early phase analgesic trials. WHY IS PAIN RESEARCH SO IMPORTANT? WHAT IS PAIN AWARENESS MONTH?

Clinical Trials

The Pharma Data

AUGUST 19, 2021

Oral presentation at the International Association for the Study of Lung Cancer’s (IASLC) 2021 World Conference on Lung Cancer (WCLC) shows evidence that the bispecific mechanism of action for RYBREVANT TM can provide anti-tumor activity against either EGFR-mutated or MET-mutated non-small cell lung cancer. 4] , [5] , [6].

Treatment

The Pharma Data

JULY 29, 2021

Today at the Alzheimer’s Association International Conference © (AAIC © 2021), Eli Lilly and Company (NYSE: LLY) presented two new exploratory analyses of data from the Phase 2 TRAILBLAZER-ALZ study. Food and Drug Administration (FDA) had granted Breakthrough Therapy designation for donanemab based on the Phase 2 data.

Disease

The Pharma Data

NOVEMBER 26, 2020

an international research-focused healthcare Group (Chiesi Group), today announced that the U.S. Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. About Pegunigalsidase Alfa.

Disease

The Pharma Data

DECEMBER 29, 2020

an international research-focused healthcare Group (Chiesi Group), today announced final study results from the BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea.

Clinical Trials

The Pharma Data

OCTOBER 18, 2020

Several clinical sites are screening patients for the Phase 1 a/b multicenter, open-label, dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the maximum tolerated dose or recommended dose in patients with relapsed or refractory AML. About CG-806.

Clinical Trials

The Pharma Data

DECEMBER 15, 2020

The clinical responses were sustained by maintenance therapy with belimumab, an antibody to B-cell activating factor. The secondary outcome involves pharmacokinetic endpoints. 2 g/dl on three consecutive available visits during the 24-week treatment period, with the response not being attributed to rescue therapy.

Disease

New Drug Approvals

SEPTEMBER 13, 2024

14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 2] Aticaprant is taken by mouth. [1]

Treatment

The Pharma Data

SEPTEMBER 8, 2021

today announced that data from its migraine portfolio will be presented at the International Headache Congress 2021, held jointly this year by the International Headache Society and the European Headache Federation, from September 8-12. Related Peptide Monoclonal Antibody Therapy Added to OnabotulinumtoxinA Treatment.

Doctors

The Pharma Data

JULY 27, 2021

Each of these three investigational therapies uses a different approach to treat a highly prevalent disease for which new treatment options are very much needed. We develop product candidates internally and through licensing collaborations, partnerships and joint ventures. ” In June 2021, AbbVie Inc.

Production

The Pharma Data

JULY 11, 2021

For more than a decade, we’ve listened to the HAE community to further understand the need for long-term, preventive targeted therapies and have committed our resources to developing treatment options,” said Neil Inhaber, M.D., There were no differences in international normalised ratio (INR) between treatment groups. Interactions.

Treatment

The Pharma Data

AUGUST 29, 2020

Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab. To date, after at least six months of stable LNP023 add-on therapy, and at the investigators’ discretion, seven patients (70%) have discontinued eculizumab and remained on LNP023 as monotherapy.

Treatment

The Pharma Data

JULY 22, 2021

Takeda Pharmaceutical Company Limited ( TSE: 4502 /NYSE:TAK) (“Takeda”) today announced the results of a phase 3 trial investigating the efficacy and safety of recombinant von Willebrand factor (rVWF) prophylaxis, 1 one of the 12 abstracts being presented at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress 2021.

Treatment

Agency IQ

AUGUST 16, 2024

However, the agency can describe a concomitant therapy in labeling for a product it has authority to regulate if it is essential for the therapeutic effect. While the ability to describe and specify aspects of the therapy is limited, the agency can specify the number and licensure of therapists who would participate in the MDMA treatment.

FDA

The Pharma Data

NOVEMBER 4, 2020

Food and Drug Administration (FDA) Breakthrough Therapy and Fast Track Designations, is now in pivotal testing, and CTP-692 for schizophrenia is currently on track for topline data readout in the first quarter of 2021,” said Roger Tung, Ph.D., and Canada) or (484) 365-2865 (International) five minutes prior to the start time.

Pharmaceuticals

The Pharma Data

DECEMBER 13, 2020

The Phase 2b trial will also evaluate efficacy on other histology endpoints (fibrosis), assessment of metabolic and non-metabolic parameters, pharmacokinetic assessment as well as safety and tolerability. Currently no curative or specific therapies are available. The Phase 2b trial is expected to begin during the second half of 2021.

Trials

The Pharma Data

OCTOBER 27, 2020

In October 2020 , we completed a second clinical study in healthy volunteers to compare the pharmacokinetics of Treprostinil Technosphere to Tyvaso. The phase 3 ADVANCE OUTCOMES study (NCT03626688) seeks to evaluate approximately 700 PAH patients, randomized 1:1 between oral ralinepag and placebo along with background therapy.

FDA

The Pharma Data

JULY 21, 2021

These data were shared as a late-breaking oral presentation during the virtual 11 th International AIDS Society Conference on HIV Science (IAS 2021) and are a follow-up to the interim analysis that was presented earlier this year at the virtual 2021 HIV Research for Prevention Conference (HIVR4P 2021).

Clinical Trials

The Pharma Data

MARCH 9, 2022

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi ® ) (STO:SOBI) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ? 12 years of age with severe hemophilia A.

Treatment

The Pharma Data

JANUARY 21, 2021

Prior to initiating treatment with CABENUVA, oral cabotegravir (VOCABRIA) and oral rilpivirine (EDURANT ® ) should be administered for approximately one month to assess the tolerability of each therapy. CABENUVA, a co-packaged kit with two injectable medicines, offers people living with HIV a new approach for maintaining viral suppression.

FDA Approval

The Pharma Data

APRIL 27, 2021

Mobocertinib is the first oral therapy specifically designed to selectively target EGFR Exon20 insertion mutations. The review is being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence (OCE), which provides a framework for concurrent submission and review of oncology products among international partners.

FDA

The Pharma Data

MARCH 9, 2021

The primary endpoint was the maximum change in plasma HIV-1 RNA during parts one and two while secondary endpoints measured safety, tolerability, and pharmacokinetic (PK) parameters. As a result, in part two, participants received GSK’254 40 mg, 80 mg, 140 mg, or placebo for a shortened period of seven days. log 10 and -2.0

Treatment

The Pharma Data

MARCH 17, 2021

Gallen International Breast Cancer Conference. Gallen International Breast Cancer Conference Poster Awards 2021. Accepted as ePoster – Adjuvant Systemic Therapy. Gallen International Breast Cancer Conference Poster Awards 2021. Accepted as ePoster – Adjuvant Systemic Therapy. The abstracts presented at St.

Trials