New Drug Approvals

JULY 7, 2025

1] Pimicotinib is under investigation in clinical trial NCT05804045 (Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)). 1] Pimicotinib is under investigation in clinical trial NCT05804045 (Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)). 6] The U.S. 6] The U.S. .

Small Molecule

New Drug Approvals

JULY 10, 2025

“Sebetralstat (KVD900): A Potent and Selective Small Molecule Plasma Kallikrein Inhibitor Featuring a Novel P1 Group as a Potential Oral On-Demand Treatment for Hereditary Angioedema” Journal of Medicinal Chemistry. Retrieved 9 July 2025 – via Business Wire. October 2022). 65 (20): 13629–13644. doi : 10.1021/acs.jmedchem.2c00921.

FDA

New Drug Approvals

APRIL 18, 2025

gmol 1 3D model ( JSmol ) Interactive image show SMILES show InChI Prete A, Auchus RJ, Ross RJ: Clinical advances in the pharmacotherapy of congenital adrenal hyperplasia. External links “Crinecerfont (Code C174708)” NCI Thesaurus.

FDA

BioPharma Drive: Drug Pricing

JULY 14, 2025

Published July 14, 2025 Ben Fidler Senior Editor post share post print email license Bain Capital, Kohlberg and Mubadala joined to invest in biopharmaceutical CDMO PCI Pharma on July 14, 2025. PCI helps biopharmaceutical companies manufacture and package drug products used in clinical trials as well as commercially.

Production

Drug Hunter

APRIL 20, 2023

The molecule was the first allosteric TYK2 inhibitor to enter clinical trials, is the first JAK-family kinase inhibitor approved for psoriasis, and is also the first approved drug targeting a pseudokinase domain. MRTX1133 is currently in Ph I/II trials as an oral agent. #3: LP0200 has completed a Ph.

Small Molecule

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. The lead candidate, HTL0022562, has advanced through preclinical development demonstrating promising and differentiated properties for further investigation in human trials. Vlad Coric , M.D.,

Small Molecule

The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. NYSE and TASE: TEVA) and MODAG GmbH today announced a strategic collaboration on the exclusive worldwide licensing and development of MODAG’s lead compound anle138b and a related compound, sery433.

Licensing

LifeSciVC

JULY 6, 2023

Although these results cannot prove that there is no possibility of finding a high-affinity small molecule binder of NBD1, they are discouraging…” It turns out the small molecules they were looking for are not only possible – they were ready for a dedicated team to push them ahead.

Small Molecule

LifeSciVC

JANUARY 16, 2024

Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Small molecule GLP1s? The incretins as a class also look interesting across a number of follow-on indications, as evidenced by clinical trials in these areas (below).

Small Molecule

The Pharma Data

JULY 18, 2021

Food and Drug Administration (FDA) granted Priority Review designation for the Biologics License Application (BLA) for their mRNA vaccine to prevent COVID-19 in individuals 16 years of age and older. The Pfizer-BioNTech COVID-19 vaccine has not been approved or licensed by the U.S. Pfizer Inc.

Licensing

The Pharma Data

MAY 7, 2021

Pfizer and BioNTech initiated the BLA by submitting the nonclinical and clinical data needed to support licensure of the COVID-19 vaccine for use in individuals 16 years of age and older. The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S.

Licensing

LifeSciVC

APRIL 24, 2024

In the absence of a clinical trial result or FDA label to point to, how does one create the case and target product profile (TPP) around a new target? and whether a molecule’s pharmacology can help to mitigate safety risk. Finally, as part of a broader pathway analysis, consider potential implications for selectivity.

Production

The Premier Consulting Blog

NOVEMBER 20, 2024

In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial small molecule or biologic product to expedite product availability for patients. The quality of data from Stage I validation activities. Prior PPQ knowledge and experience with similar products.

Small Molecule

The Pharma Data

DECEMBER 13, 2020

RLY-1971 is a potent small molecule inhibitor of Src homology region 2 domain-containing phosphatase-2 (SHP2). RLY-1971 is currently being evaluated in a first-in-human trial designed to treat patients with advanced or metastatic solid tumors. To learn more about the first-in-human clinical trial of RLY-1971, please visit here.

Licensing

New Drug Approvals

JUNE 26, 2025

Acoltremon AR-15512 CAS 68489-09-8 WeightAverage: 289.419 Monoisotopic: 289.204179113 Chemical FormulaC 18 H 27 NO 2 FDA 2025, 5/28/2025, To treat the signs and symptoms of dry eye disease Tryptyr WS-12 WS 12 (1R,2S,5R)-N-(4-methoxyphenyl)-5-methyl-2-(propan-2-yl)cyclohexane-1-carboxamide Fema No. Archived from the original on 29 May 2025.

FDA

The Pharma Data

DECEMBER 15, 2020

SetPoint Medical received FDA Investigational Device Exemption (IDE) approval for a multicenter, double-blind, randomized, sham-controlled pivotal trial that will enroll up to 250 patients at 40 clinical trial sites in the U.S. Small Molecule Inhibitors. Oral, Small Molecules.

Disease

The Pharma Data

SEPTEMBER 12, 2020

Its broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor T cells, bi-specific checkpoint immuno-modulators, targeted cancer antibodies and small molecules.

Vaccine

The Pharma Data

NOVEMBER 18, 2020

Its mission is to develop and license novel therapeutic agents for unmet medical needs against cancer targets such as Globo Series (including Globo H, SSEA-3 and SSEA-4), AKR1C3, and other promising targets. The above poster presentations will be available online at www.obipharma.com on November 23, 2020. About OBI Pharma. OBI Pharma, Inc.,

Vaccine

The Pharma Data

OCTOBER 18, 2020

Berk served as an investigator on several industry-sponsored and cooperative group clinical trials, including the pivotal trials for Gleevec® and Avastin®. Inflection Biosciences Ltd is developing small molecule therapeutics for the treatment of cancer. About Inflection Biosciences.

Small Molecule

New Drug Approvals

DECEMBER 16, 2023

“Small-molecule factor B inhibitor for the treatment of complement-mediated diseases” Proceedings of the National Academy of Sciences of the United States of America. 1 Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase. 5 December 2023.

FDA

The Pharma Data

OCTOBER 14, 2020

Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.

Pharmaceuticals

The Pharma Data

DECEMBER 11, 2020

Pfizer and BioNTech expect to file a Biologics License Application for possible full regulatory approval in 2021. The participants in our COVID-19 vaccine clinical trial are courageous volunteers who have made a personal and important choice to help make a difference during this pandemic. About the Phase 2/3 Study.

Vaccine

The Pharma Data

OCTOBER 26, 2020

The mAb portfolio is complemented by a non-antibiotic novel mechanism small molecule anti-infective candidate being developed to treat lung infections in cystic fibrosis patients. AR-711 is an in-licensed mAb that is directed against the receptor binding domain of the SARS-Cov 2 virus. AR-711 (COVID-19). AR-201 (RSV infection).

Pharmaceuticals

The Pharma Data

MAY 6, 2021

The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Severe allergic reactions have been reported following the Pfizer-BioNTech COVID-19 Vaccine during mass vaccination outside of clinical trials.

Vaccine

The Pharma Data

NOVEMBER 4, 2020

Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. The THRIVE-AA1 Phase 3 trial is a randomized, double-blind, placebo-controlled clinical trial of CTP-543 to evaluate hair regrowth using the Severity of Alopecia Tool (SALT) after 24 weeks of dosing in approximately 700 adult patients with moderate to severe alopecia areata.

Pharmaceuticals

The Pharma Data

SEPTEMBER 9, 2020

“We have activated our supply chain, most importantly our site in Belgium, and are starting to manufacture so that our vaccine would be available as soon as possible, if our clinical trials prove successful and regulatory approval is granted.”. “As billion doses by the end of 2021.

Vaccine

The Pharma Data

JULY 21, 2021

The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. myocarditis and pericarditis. Additional adverse reactions, some of which may be serious, may become apparent with more widespread use of the Pfizer-BioNTech COVID-19 Vaccine.

Vaccine

The Pharma Data

JANUARY 26, 2021

Food and Drug Administration’s Office of Orphan Products Development has granted Orphan Drug Designation to Uttroside-B , a small molecule chemotherapeutic for the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer. (OTCQB: QBIO), announced today that the U.S. billion by 2027.

Treatment

The Pharma Data

NOVEMBER 20, 2020

Pfizer and BioNTech are extremely grateful to the study volunteers and investigative site staff in the clinical trial program, as their involvement was crucial to today’s important milestone in the companies’ efforts to address the COVID-19 global pandemic. as well as Europe, Latin America, and South Africa. About the Study.

Vaccine

The Pharma Data

SEPTEMBER 9, 2020

The study is an event-driven trial. Pfizer and BioNTech are committed to decreasing health disparities in underrepresented populations through the clinical trial process. Additional enrollment is planned in Germany, Turkey and South Africa.

Vaccine

The Pharma Data

FEBRUARY 24, 2022

The positive opinion adopted by the CHMP is based on an evaluation of interim safety and efficacy data from a clinical trial of a booster dose of the vaccine in those aged 16 and over, together with published literature and post authorisation data plus real-world evidence from the use of booster doses in young patients in Israel.

Vaccine

The Pharma Data

JANUARY 10, 2021

The company submitted its Biologic License Application to the FDA which has set a Prescription Drug User Fee Act (PDUFA) date for June 25, 2021. The TransCon technology platform can be applied broadly to proteins, peptides or small molecules in multiple therapeutic areas, and can be designed for systemic or localized release.

Clinical Trials

The Pharma Data

NOVEMBER 5, 2020

We believe the target product profiles of our two ophthalmic candidates, Nyxol and APX3330, collectively studied in 18 clinical trials and each having market potential, create an opportunity for Ocuphire to become a leading ophthalmic company focused on improving vision and clarity. Cantor Fitzgerald & Co.

Clinical Trials

The Pharma Data

AUGUST 20, 2020

The Phase 2/3 trial enrollment to date has exceeded 11,000 participants with a second dose underway. Pfizer and BioNTech are committed to decreasing health disparities in underrepresented populations through the clinical trial process.

Vaccine

The Pharma Data

NOVEMBER 9, 2020

Clinical trial to continue through to final analysis at 164 confirmed cases in order to collect further data and characterize the vaccine candidate’s performance against other study endpoints. Pfizer Inc. The data will be discussed with regulatory authorities worldwide. “I

Vaccine

The Pharma Data

OCTOBER 14, 2020

This work may also guide the selection of appropriate participants for clinical trials of COVID-19 vaccines, including future human trials using Tonix’s TNX-1800, a live replicating, attenuated COVID-19 vaccine candidate designed to confer durable T cell immunity.”. President and Chief Executive Officer of Tonix Pharmaceuticals.

Vaccine

New Drug Approvals

APRIL 30, 2025

“A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia” Trials. A clinical trial to test amlodipine as a new treatment for vascular dementia. 1] It is the L -form of acetylleucine.

FDA