New Drug Approvals

JULY 25, 2025

Corticosteroid therapy is the current standard of care for DMD despite relatively high rates of adverse effects. 79 (2022) 1005–1014. [71] 75,76 This also results in decreased glucocorticoid receptor-drive transactivation, ultimately improving the safety profile of vamorolone as compared to other corticosteroid therapies.

FDA

BioPharma Drive: Drug Pricing

JUNE 11, 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. Among those who had gotten another such therapy, the rates were 52% and 62% in those trials.

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] PATENTS EP3643715 WO2018233527 US20200140431 US11180495 WO2018233527 US20200140431 References ^ Vaynrub A, Healey JH, Tap W, Vaynrub M (2022). 6] The U.S. 15 : 53–66. PMC 8763255.

Small Molecule

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). 1 November 2022). 17 February 2022). 1] [2] [5] Names Crinecerfont is the international nonproprietary name.

FDA

New Drug Approvals

APRIL 19, 2025

21 December 2022. 24 August 2022. New Drug Therapy Approvals 2024 (PDF). “Metoprolol Therapy and CYP2D6 Genotype” In Pratt VM, McLeod HL, Rubinstein WS, et al. Further reading Shiga T (June 2022). . ^ “Details for: Sibboran” Health Canada. 20 November 2023. Retrieved 2 April 2024.

FDA

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

Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies

Drug Hunter

JUNE 25, 2023

This edition includes Maze’s glycogen synthase 1 (GYS1) inhibitors that were recently licensed to Sanofi, allosteric androgen receptor (AR) modulators that may be of interest to targeted protein degradation researchers, and brain-penetrant HER2 and ROCK2 inhibitors.

Targeted Protein Degradation

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

The Pharma Data

DECEMBER 13, 2020

Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced that the company conducted a Type B Meeting for omidubicel with the U.S. The revised plan could support a potential commercial launch of omidubicel in the United States as early as mid-2022.

Licensing

The Pharma Data

JUNE 14, 2023

With our oncology strategy, we are dedicated to accelerating the research and development of novel cancer therapies that have the potential to make a meaningful difference in the lives of patients.” In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. billion euros. Source link: [link]

Licensing

FDA Law Blog: Drug Discovery

JANUARY 4, 2023

Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and contr­­ols (CMC).

Therapies

Drug Target Review

APRIL 4, 2024

The animal model is based on creating a dopamine-deficient state, by a specific lesion of dopaminergic cells, and then exposing it to repeated substitution therapy with L-DOPA. Nicholas served as the CEO of IRLAB Sweden between 2012 and 2022 and for IRLAB therapeutics 2016-2022. In 2013, Nicholas co-founded IRLAB Sweden.

Disease

LifeSciVC

JANUARY 16, 2024

By Aimee Raleigh, Principal at Atlas Venture, as part of the From The Trenches feature of LifeSciVC Just in time for new years’ reflections and resolutions, this year’s JPM felt like a refreshing burst of enthusiasm for a sector that has seen its challenges in 2022 and 2023 but also some green shoots. Join the club.

Small Molecule

The Pharma Data

JANUARY 27, 2021

. – Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. “We

Therapies

DrugBank

OCTOBER 4, 2024

This involves assessing the strength and breadth of patents, evaluating the potential for future patent challenges, and analyzing the value derived from licensing agreements and royalty streams. GlaxoSmithKline's spin-off of its consumer healthcare division , Haleon, in 2022 is a case in point.

Pharmaceuticals

The Pharma Data

NOVEMBER 25, 2020

The majority of the work will be completed in 2021 to start clinical trials as early as possible during 2022. The main project, the antibody CAN04, is being studied clinically as combination therapy with chemotherapy or immune therapy with a primary focus on non-small cell lung cancer and pancreatic cancer. About BioInvent.

Clinical Trials

The Premier Consulting Blog

OCTOBER 21, 2024

Accelerated approval is an expedited regulatory pathway designed to hasten the availability of drugs (including biologics) that treat serious conditions, offer advantages over existing therapies, and address unmet medical needs. This approach ensures timely completion of confirmatory trials in closer proximity to the initial approval.

Trials

The Pharma Data

JULY 22, 2021

We share Pfizer’s deep commitment to people with breast cancer and are thrilled to partner with them to develop this potentially best-in-class therapy. Endocrine therapy is a backbone of ER+ breast cancer treatment and is used as monotherapy or as combination therapy as a standard of care across treatment settings.

Targeted Protein Degradation

BioPharma Drive: Drug Pricing

JULY 23, 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. Seven of these CAR-T therapies are available in the U.S., You can unsubscribe at anytime.

Therapies

New Drug Approvals

DECEMBER 24, 2023

“Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen” Neurology and Therapy. December 2022). . | Facebook Twitter FACEBOOK join me on twitter Anthony Melvin Crasto Dr. | twitter +919321316780 call whatsaapp EMAIL.

Clinical Trials

The Pharma Data

APRIL 19, 2022

tumour response rate If approved, Enhertu to provide patients with a much-needed targeted therapy option. 1 The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is during the third quarter of 2022. The application has also been granted Priority Review.

Trials

FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

The statutory Accelerated Approval provisions were amended in 2023 as part of the Food and Drug Omnibus Reform Act of 2022 (“FDORA”) to, among other things, give FDA greater authority to expedite the withdrawal of approval of an Accelerated Approval product if clinical benefit is not confirmed post-NDA or -BLA approval. FDA-2022-E-3124 ).

FDA

The Pharma Data

JANUARY 18, 2021

The European sales and marketing infrastructure is being put in place to be ready for commercial launch as early as the beginning of 2022. At the same time, we continue to prepare for the filing of the Biologics License Application with the U.S. Temporary Authorization for Use (ATU) of LUMEVOQ ®. GenSight Biologics S.A.

Treatment

The Pharma Data

APRIL 27, 2023

It is also an off-the-shelf therapy, meaning that people do not have to wait for cell collection and genetic engineering before starting treatment, which could be particularly important for patients who are at a high-risk of their disease progressing. “New Columvi has the potential to change the current standard of care in DLBCL.

Therapies

The Pharma Data

MARCH 27, 2022

In February 2022, the U.S. European Union and Japan in 2022. “In 2022, OLUMIANT could become the first medicine ever approved to treat adults with alopecia areata. OLUMIANT, a once-daily, oral JAK inhibitor was discovered by Incyte and licensed to Lilly. Lilly expects regulatory decisions in the U.S.,

Licensing

The Pharma Data

NOVEMBER 24, 2020

Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. The study aims to conclude in the beginning of 2022.

FDA

Drug Target Review

JUNE 3, 2024

Results from these preclinical studies were published in the peer-reviewed publication, Journal of Immunology in August 2022 as a “Top Read” article. Could you explain the development of CNP-108 for T cell immunogenicity in gene therapy, and how it induces the immune system to build a tolerance to gene therapy treatments?

Disease

The Pharma Data

MAY 2, 2022

Under the terms of the settlement agreement, the litigation between the parties in the United States District Court for the District of New Jersey will be ended, and Lupin will have a license to sell its generic product beginning April 2033, or earlier under certain circumstances. On March 9, 2022, the U.S.

Pharmaceuticals

The Pharma Data

JULY 21, 2021

Biovac will obtain drug substance from facilities in Europe, and manufacturing of finished doses will commence in 2022. The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. 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 23, 2021

These doses are expected to be delivered from October 2021 through April 2022. The companies expect to deliver 110 million of the additional doses by December 31, 2021, with the remaining 90 million doses to be delivered no later than April 30, 2022. The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S.

Vaccine

The Pharma Data

JANUARY 10, 2021

Depending on the development of the escalating COVID-19 pandemic in the United States and on the resulting impact on enrollment, the Company expects to complete enrollment of this study in 2022 enabling a potential Biologics License Application (BLA) submission by 2023 under the accelerated approval pathway.

Therapies

New Drug Approvals

OCTOBER 24, 2023

“Etrasimod as induction and maintenance therapy for ulcerative colitis (ELEVATE): two randomised, double-blind, placebo-controlled, phase 3 studies” Lancet. July 2022). . | Facebook Twitter FACEBOOK join me on twitter Anthony Melvin Crasto Dr. | twitter +919321316780 call whatsaapp EMAIL. 401 (10383): 1159–1171.

FDA

Agency IQ

JANUARY 12, 2024

FDA received its most recent statutory authority to collect fees through PDUFA VII on September 30, 2022, when it passed user fee reauthorization as part of the Continuing Resolution to fund the federal government. According to Downey, the timeline of pre-license inspections for biologics can create challenges.

FDA

The Pharma Data

DECEMBER 30, 2020

31, 2020 /PRNewswire/ — ImmVira Group Company (“the Company”), a biotechnology platform dedicated to the development of oncolytic virus(“OV”) and vector type approaches to create more effective and safer therapies against cancer, announced today the signing of Series C financing.

Virus

The Pharma Data

OCTOBER 27, 2020

Nasdaq: MDWD), a fully-integrated biopharmaceutical company bringing innovative therapies to address unmet needs in severe burn and wound management, today announced it has completed the enrollment stage of its NexoBrid ® Phase 3 pediatric clinical study (CIDS – Children Innovation Debridement Study). “We YAVNE, Israel, Oct.

Clinical Trials

New Drug Approvals

DECEMBER 16, 2023

August 2022). . | Facebook Twitter FACEBOOK join me on twitter Anthony Melvin Crasto Dr. | twitter +919321316780 call whatsaapp EMAIL. 5 December 2023. . ^ [link] Archived 10 December 2023 at the Wayback Machine This article incorporates text from this source, which is in the public domain. 6 (15): 4450–4460. doi : 10.1182/bloodadvances.2022006960.

FDA