The Pharma Data

AUGUST 1, 2025

PRIME allows us to align early with the EMA on key evidence requirements and potentially accelerate our path to marketing authorization of BGB-16673 for patients with relapsed or refractory Waldenstrom’s macroglobulinemia.” There remains a high unmet need for more effective and durable therapies, especially in the post-BTK inhibitor setting.

Targeted Protein Degradation

Drug Target Review

JUNE 2, 2025

Bioconjugates are rapidly emerging as a key frontier in targeted therapies, particularly through Antibody-Drug Conjugates (ADCs) , which combine antibodies with cytotoxic drugs to deliver more precise and effective treatments. Very often, you have a payload that is extremely toxic, and then you require specially trained people to handle it.

Small Molecule

Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments.

Drugs

Pharma Manufacturing

JUNE 17, 2025

Dubey estimates that there are only a little more than 15 drug products in the market today that are being manufactured using continuous manufacturing. “The ones that don’t go through the FDA are made for local markets in India and China. “Those are through the FDA,” he said.

RNA

BioPharma Drive: Drug Pricing

JULY 8, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.

Targeted Protein Degradation

The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

Treatment

Drug Patent Watch

JANUARY 13, 2025

As we delve into the intricate world of biosimilar market dynamics, we’ll explore how these complex molecules are reshaping treatment paradigms across diverse patient populations. ”[1] The global biosimilars market is experiencing exponential growth, with projections indicating it will reach $69.4

Biosimilars

The Pharma Data

JUNE 24, 2025

This expanded partnership reflects Bayer’s ongoing commitment to strengthening its presence in China—home to the world’s second-largest pharmaceutical market—and underscores its strategic vision to bolster healthcare innovation through cross-sector collaboration.

Pharma R&D

LifeSciVC

JULY 8, 2025

This growing complexity gives rise to a deeper set of questions, ones that extend beyond infrastructure and markets. One team might be working on a cell therapy, another on RNA, another on small molecules. These organizations rarely follow a single asset path.

Hospitals

Codon

JULY 28, 2025

The gene therapy is designed to protect this infant from the neurological disorders that otherwise would have resulted from this rare, and often fatal, condition. And while matching a gene therapy to its targeted mutation is an intricate and nuanced process, much boils down to two core elements: a vector and its cargo.

Immune Response

New Drug Approvals

APRIL 18, 2025

2] The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track , breakthrough therapy , orphan drug , and priority review designations. [2] New Drug Therapy Approvals 2024 (PDF). 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2]

FDA

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. The global ADC market is expected to continue this steep upward trajectory and is anticipated to exceed $16 Billion by 2026. 3D rendering of Antibody Drug Conjugate Molecules.

Small Molecule

Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. For manufacturing, it’s no different.

Therapies

ASPET

SEPTEMBER 7, 2023

Avalglucosidase alfa has received marketing authorization in several countries for infantile-onset and/or late-onset Pompe disease. This recently approved enzyme replacement therapy (ERT) was glycoengineered to maximize CIMPR binding through high-affinity interactions with ~7 bis-M6P moieties.

Small Molecule

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.

Therapies

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

Small Molecule

DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

Drug Development

The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS in December 2022 about changes to their initial 2017 partnership whereby Daiichi Sankyo obtained the Marketing Authorization for Yescarta ®. to Gilead Sciences K.K.,

Therapies

The Pharma Data

SEPTEMBER 10, 2021

– Abrocitinib is a once-daily oral JAK1 inhibitor indicated in Great Britain for the treatment of moderate to severe atopic dermatitis in patients aged 12 years and over, who are candidates for systemic therapy-. This is the first marketing authorization globally for abrocitinib-. About CIBINQO ® (abrocitinib).

Marketing

Drug Target Review

JULY 23, 2024

This laid the groundwork for my career in drug discovery using novel modalities like small molecule inhibitors, antibodies, RNA interference (RNAi) and, currently, small molecule protein degraders – which are opportunities to change how we think about medicines.

Small Molecule

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. We look forward to working on this new collaboration with Biohaven, a world leader in the clinical development of CGRP-targeted therapies.” TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,

Small Molecule

The Premier Consulting Blog

NOVEMBER 20, 2024

For drugs with FDA Orphan Drug Designation (ODD), Breakthrough Therapy Designation (BTD), and EMA PRIority MEdicines designation (PRIME), all clinical, non-clinical, and CMC requirements are still required for regulatory approval, despite being on pathways for an expedited approval process.

Small Molecule

LifeSciVC

APRIL 24, 2024

and whether a molecule’s pharmacology can help to mitigate safety risk. Especially for oligo or small molecule discovery and development, it’s important to understand whether there are highly homologous sequences or proteins that may be impacted by a given therapeutic approach. in liver, in CNS)? Consider Fig.

Production

Conversations in Drug Development Trends

APRIL 4, 2024

Stability Tests: For small molecule, only -20°C stability testing is required, while large molecule need both -20°C and -70°C tests if samples are intended for storage at these temperatures. Select the right partner with experience meeting regulatory standards for all target markets.

Small Molecule

The Pharma Data

AUGUST 7, 2021

Vividion’s platform is able to produce a variety of small molecule therapies across indications, with initial focus on targets relevant to oncology and immunology. The acquisition of Vividion strengthens Bayer’s small molecule capabilities and expands Bayer’s reach into new modalities.

Small Molecule

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

The Pharma Data

JANUARY 25, 2021

–( BUSINESS WIRE )– Veralox Therapeutics, a biotechnology company developing first-in-class small molecule therapeutics that treat the underlying pathologies of diseases with significant unmet medical needs, today announced that the U.S. 26, 2021 13:00 UTC. FREDERICK, Md.–( About VLX-1005.

FDA

Creative Biolabs

JUNE 29, 2023

At present, a large number of G protein-coupled receptor-targeted drugs have been marketed, and a large number of drugs are under development. While the development of small molecule drugs targeting GPCRs, the development of monoclonal antibody drugs has also received great attention.

Therapies

The Pharma Data

NOVEMBER 15, 2020

As its name aptly suggests, Urovant is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for urologic conditions such as overactive bladder (OAB) and maladies involving adjacent areas of the human anatomy. .

Science

PPD

OCTOBER 18, 2023

There has been an unprecedented expansion of different types of pharmaceutical therapies, as well as a change in the overall approach to health care. Continued expansion of the biotech industry is occurring in parallel, with an estimated compound annual growth rate of 13.96% from 2023-2030.

Laboratories

The Pharma Data

NOVEMBER 11, 2020

As an orally bioavailable small molecule with broad antiviral activity, SLV213 could be a valuable treatment to meet today’s urgent need to fight COVID-19 as well other life-threatening infectious diseases, such as Chagas disease, Ebola virus disease, and Nipah virus infection.”. About SLV213.

Treatment

The Pharma Data

APRIL 6, 2021

Approvals were based on the overall response rate (ORR) from the open-label, single-arm Phase II CHRONOS-1 (NCT01660451) trial of copanlisib monotherapy in 104 adult patients with follicular B-cell NHL who had relapsed disease following at least two prior systemic therapies. PSMA-TTC is currently being investigated in Phase I studies.

Research

Alta Sciences

AUGUST 29, 2024

Listen or read Issue 33 of The Altascientist on altasciences.com , or wherever you listen to podcasts: GROWING NEEDS FOR CNS THERAPY SOLUTIONS The different parts of the nervous system, including the brain and spinal cord and the peripheral nervous system, are important drug targets for many serious diseases affecting human health.

Drug Development

The Pharma Data

SEPTEMBER 8, 2021

Adds Rezurock™ (belumosudil) an FDA-approved, first-in-class treatment for adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. Sanofi has entered into a definitive merger agreement with Kadmon Holdings, Inc. Transaction Terms.

FDA Approval

The Pharma Data

OCTOBER 6, 2021

The European Commission (EC) will review the CHMP recommendation and is expected to make a final decision on a variation to the Conditional Marketing Authorization in the near future. At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives.

Vaccine

The Pharma Data

JULY 18, 2021

BioNTech is the Marketing Authorization Holder in the European Union, and the holder of emergency use authorizations or equivalent in the United States (jointly with Pfizer), Canada and other countries in advance of a planned application for full marketing authorizations in these countries.

Licensing

The Pharma Data

JANUARY 13, 2021

Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small-molecule treatments for developmental and epileptic encephalopathies, other epilepsies, neuropathic pain, and tinnitus.

Trials