New Drug Approvals

JULY 10, 2025

1] The US Food and Drug Administration granted the application for sebetralstat orphan drug designation. [4] “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.

FDA

New Drug Approvals

APRIL 18, 2025

2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 1] [2] Adverse effects The US Food and Drug Administration prescription label for crinecerfont has a warning for acute adrenal insufficiency or adrenal crisis. [2] Food and Drug Administration (FDA) (Press release). January 2025.

FDA

New Drug Approvals

JULY 7, 2025

History In December 2023, Abbisko Therapeutics entered into a licensing agreement for pimicotinib in all indications for China rights with Merck KGaA. [2] History In December 2023, Abbisko Therapeutics entered into a licensing agreement for pimicotinib in all indications for China rights with Merck KGaA. [2] 6] The U.S. 6] The U.S.

Small Molecule

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. drug production.

Production

Drug Hunter

APRIL 20, 2023

We asked the global drug discovery community to nominate and vote on their favorite molecule from 2022, and the results are in. Here, we highlight what makes deucravacitinib so impressive to the drug discovery community. Here, we highlight what makes deucravacitinib so impressive to the drug discovery community.

Small Molecule

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. Chief Executive Officer of Biohaven, said: “We are excited to enter into this agreement with Sosei Heptares, a world leader in GPCR drug discovery. TOKYO and CAMBRIDGE, England , Dec. 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

But they struggled to find a drug candidate that could bind to and stabilize it. After years of steady progress and tens of millions of dollars invested, Sanofi Genzyme corporate strategy shifted, and eventually a strategic decision was made to suspend the program, focus resources on immunology/immuno-oncology, and out license these assets.

Small Molecule

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. It ensures that a production process consistently yields products of predetermined quality and safety.

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 Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is in January 2022.

Licensing

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? Whether trial design, execution, or otherwise, drug development even where there is precedent is a challenging road and should not be taken for granted.

Small Molecule

LifeSciVC

APRIL 24, 2024

Is a novel target at the inflection point where enough evidence is available to suggest it may prove to be a compelling drug? and whether a molecule’s pharmacology can help to mitigate safety risk. In order to start building a case for or against a target, I like to start with genetics – first human and then mouse. in liver, in CNS)?

Production

The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. This is because many proteins do not have small-molecule binding sites. The first identifies the 3D RNA motifs.

RNA

The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial.

Disease

The Pharma Data

MAY 7, 2021

The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Food and Drug Administration (FDA), but has been authorized for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19) for use in individuals 16 years of age and older.

Licensing

The Pharma Data

DECEMBER 13, 2020

RLY-1971 is a potent small molecule inhibitor of Src homology region 2 domain-containing phosphatase-2 (SHP2). Relay Therapeutics (Nasdaq: RLAY) is a clinical-stage precision medicines company transforming the drug discovery process with the goal of bringing life-changing therapies to patients. . CAMBRIDGE, Mass.,

Licensing

The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. Bayer today announced that it has entered into an agreement to acquire Noria Therapeutics Inc. Noria) and PSMA Therapeutics Inc.

Small Molecule

New Drug Approvals

OCTOBER 12, 2024

Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. Years later, a subgroup analysis of the trial data indicated a potential positive effect in participants who carried two copies of ApoE4 ( Abushakra et al., 2016 ; Abushakra et al., Hey, et al. 2018; 32(9): 849–861. [2]. Hey JA, et al.

Pharmacokinetics

The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for SLV213 for the treatment of COVID-19 and has dosed the first subjects in a Phase 1 clinical study. While SLV213 can be dosed orally or intravenously, Selva is first advancing it as an oral drug candidate for COVID-19. About SLV213.

Treatment

The Premier Consulting Blog

AUGUST 8, 2024

On July 31, 2024, the US Food and Drug Administration (FDA) announced Fiscal Year 2025 (FY2025) Prescription Drug User Fee Amendments of 2022 (PDUFA VII) fee rates for the review of human drug and biological product applications along with prescription drug program fees.

Drugs

New Drug Approvals

JUNE 26, 2025

5] References ^ Jump up to: a b [link] ^ “Novel Drug Approvals for 2025” U.S. 5] References ^ Jump up to: a b [link] ^ “Novel Drug Approvals for 2025” U.S. Food and Drug Administration (FDA). 4] Society and culture Legal status Acoltremon was approved for medical use in the United States in May 2025. [5]

FDA

FDA Law Blog: Biosimilars

FEBRUARY 26, 2024

Relevant here, FDA interpreted in Guidance that a proposed injectable biosimilar must “demonstrate that its product has the same strength as the reference product by demonstrating that both products have the same total content of drug substance (in mass or units of activity) and the same concentration of drug substance.”

FDA

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. NEW YORK , Jan. 27, 2021 /PRNewswire/ — Q Biomed Inc.

Treatment

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. ELX-02 is an investigational drug that has not been approved by any global regulatory body.

Pharmaceuticals

Drug Target Review

NOVEMBER 22, 2023

Whilst TGF-β is an attractive target for cancer therapy because of its critical role in promoting tumor aggressiveness, immune escape and resistance to therapy, activation of alternative resistance pathways has hindered drug development attempts in the past.

Licensing

New Drug Approvals

DECEMBER 16, 2023

1] Iptacopan was approved by the US Food and Drug Administration (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria in December 2023. [2] 5] [6] Iptacopan is also investigated as a drug in other complement-mediated diseases, like age-related macular degeneration and some types of glomerulopathies. [7]

FDA

The Pharma Data

DECEMBER 15, 2020

Many drug makers are investing in new science and hope to develop new therapeutics that address autoimmune disease. Scientists will be testing a drug class targeting the C-reactive protein (CRP) marker of acute inflammation in the body. They’ll use software that searches available drugs, as well as drug-like compounds.

Disease

The Pharma Data

OCTOBER 18, 2020

Dr. Berk, brings over 30 years of experience in medicine, industry and academia, with extensive expertise in oncology drug development and regulatory strategy. Inflection Biosciences Ltd is developing small molecule therapeutics for the treatment of cancer. Berk, MD, to its Board of Directors. biopharma companies.

Small Molecule

The Pharma Data

AUGUST 21, 2020

Fortunately, as a member of the Novartis Business Development & Licensing (BD&L) department, she was in a position to try to help. Jennifer Leeds , Head of West Coast Search and Evaluation, NIBR Business Development & Licensing. Knowing that a pandemic was likely coming made watching the crisis unfold no less heartbreaking.

Virus

The Pharma Data

NOVEMBER 18, 2021

— Gilead Exercises Options to Arcus’s Anti-TIGIT Program (Domvanalimab and AB308), Etrumadenant (A2a/A2b Adenosine Receptor Antagonist) and Quemliclustat (Small Molecule CD73 Inhibitor) — — Arcus to Receive Option Payments Totaling $725 million — Gilead Lores,Inc. Nasdaq GILD) and Arcus Biosciences,Inc.

Research

The Pharma Data

OCTOBER 26, 2020

Such deficiencies include, but are not limited to, increasing drug resistance, short duration of efficacy, disruption of the normal flora of the human microbiome and lack of differentiation among current treatments. AR-711 is an in-licensed mAb that is directed against the receptor binding domain of the SARS-Cov 2 virus.

Pharmaceuticals

The Pharma Data

FEBRUARY 24, 2022

Food and Drug Administration (FDA) earlier this year as an expansion to the existing EUA for the primary series. The Company exploits a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. No new safety concerns were identified from the data available.

Vaccine

The Pharma Data

NOVEMBER 8, 2020

Almirall will initiate a Phase III trial soon to demonstrate the drug’s efficacy and safety in China. 10% of its Net Sales in R&D and also invests significantly in business development and in-licensing. Several novel small molecule leads were the subject of this achievement.

Licensing

New Drug Approvals

APRIL 30, 2025

1] [2] [3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C. [2] 2] The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review , fast track , orphan drug , and rare pediatric disease designations. [2]

FDA

The Pharma Data

SEPTEMBER 12, 2020

Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. NEW YORK & MAINZ, Germany–(BUSINESS WIRE)– Pfizer Inc.

Vaccine

The Pharma Data

JANUARY 10, 2021

. TransCon hGH (lonapegsomatropin) : Lonapegsomatropin is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) currently under review for use in pediatric growth hormone deficiency (GHD) by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA): .

Clinical Trials

The Pharma Data

OCTOBER 14, 2020

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. The CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions.

Vaccine