The Pharma Data

MAY 31, 2022

. “The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” said Richard Finkel, M.D., Jude Children’s Research Hospital. Food and Drug Administration in 2017.

FDA Approval

Advarra

AUGUST 16, 2022

Orphan drugs have historically faced a number of barriers, such as limited research and development (R&D) investment due to an expected lack of profitability as well as challenges in clinical trial design and recruitment. Food and Drug Administration (FDA) approval. These centers, in conjunction with the FDA and U.S.

Disease

The Pharma Data

JANUARY 5, 2021

based contract manufacturing business, Benuvia Manufacturing, which has significant chemistry and formulation capabilities, including manufacturing our FDA-approved cannabinoid drug, SYNDROS ® ,” said Todd C. We look forward to supporting Radius through our U.S. Davis, executive chairman of Benuvia. Disease Highlights.

Disease

Drug Target Review

AUGUST 29, 2024

MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs. What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies?

Therapies

Vial

DECEMBER 5, 2023

Next-generation sequencing (NGS) has revolutionized scientific research and clinical genomics due to high-throughput multiplexing and shed light on genome structure, function, and dynamics. It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development.

Clinical Trials

The Pharma Data

OCTOBER 15, 2020

The findings were presented at TCT Connect , the 32nd annual scientific symposium of the Cardiovascular Research Foundation. Researchers found an 18% relative improvement in overall survival in the recent cohort, indicating an associated benefit to placing Impella pre-PCI. This press release features multimedia. The Impella 2.5,

Hospitals

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues. Asia, and Europe.

Drug Development

Drug Target Review

JULY 5, 2024

Recently, a network-based drug-screening platform has been developed to test FDA-approved drugs for Alzheimer’s disease (AD) using 1,300 iPSC-derived organoids from 11 sporadic AD patients. 545:54-59 (2017). A significant challenge for the field is incorporating 3D models into high-throughput platforms. Molecular Autism.

Drugs

NIH Director's Blog: Drug Development

JULY 20, 2017

The first genetic mutation that causes CF was discovered by a collaborative effort between my own research lab at the University of Michigan, Ann Arbor, and colleagues at the Hospital for Sick Children in Toronto—more than 25 years ago [1]. Cystic Fibrosis Foundation News Release, July 18, 2017. First, a bit of history!

Clinical Trials

Drug Target Review

AUGUST 3, 2023

Engaging patient groups and recruiting participants for preclinical research studies remains a major challenge, as the necessary infrastructure for matchmaking and follow-up is still in its early stages. Still a big problem with today’s preclinical research methods is engaging patient groups and recruiting people to take part.

DNA

Vial

JUNE 12, 2024

Utilizing PROs enables the active participation of patients in their care outcomes – both in a clinical setting and in clinical research. While PROs may not have unconditional acceptance and wide use in clinical research across all therapeutic areas, they have been in use for over a decade in some. For HF, Savarese et al.

Clinical Trials

The Pharma Data

JANUARY 31, 2021

Clover Biopharmaceuticals is a global, clinical-stage, research-based biotechnology company focused on discovering, developing and commercializing transformative biologic therapies, with a focus on oncology and autoimmune diseases, as well as viral vaccines. About Clover Biopharmaceuticals. For more information, visit www.dynavax.com.

Clinical Trials

Drug Target Review

DECEMBER 5, 2024

Among them, adeno-associated viruses (AAVs) stand out due to their small size, high efficiency in gene delivery, low immunogenicity, and potential for customisable tissue targeting, making them the leading tool in gene expression research.

Therapies

New Drug Approvals

AUGUST 26, 2023

Zuranolone CAS 1632051-40-1 Zurzuvae FDA APPROVED 8/4/2023, To treat postpartum depression Press Release WeightAverage: 409.574 Monoisotopic: 409.272927379Chemical FormulaC 25 H 35 N 3 O 2 SAGE 217 SAGE-217 SAGE217 Zuranolone , sold under the brand name Zurzuvae , is a medication used for the treatment of postpartum depression. [1]

FDA

The Pharma Data

JUNE 18, 2021

BOTOX ® Cosmetic is the first and only product of its kind FDA-approved to treat three areas, temporarily improving the appearance of moderate to severe forehead lines, glabellar lines and crow’s feet in adults. Department of Health and Human Services FDA Approval Letter for BOTOX® Cosmetic for Glabellar Lines, 2002.

Doctors

The Pharma Data

DECEMBER 3, 2020

Clover Biopharmaceuticals is a global, clinical-stage, research-based biotechnology company focused on discovering, developing and commercializing transformative biologic therapies, with a focus on oncology and autoimmune diseases, as well as viral vaccines. About Clover Biopharmaceuticals. For more information, visit www.dynavax.com.

Vaccine

The Pharma Data

DECEMBER 15, 2020

Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. The researchers regulated the abnormal immunological memory processes found in these patients. Daratumumab already is approved for the treatment of multiple myeloma. It may be life-threatening.

Disease

Agency IQ

SEPTEMBER 15, 2023

Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDA approval so long as it adhered to pre-set terms under the monograph. In 2017, the first year of the PDUFA VI agreement under the FDA Reauthorization Act, the fees were published on September 14, 2017.

FDA

The Pharma Data

SEPTEMBER 2, 2020

New Phase III data from SAkuraStar and SAkuraSky studies demonstrate reduced severity of relapses with ENSPRYNG (satralizumab), recently FDA-approved as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD).

Clinical Trials

The Pharma Data

APRIL 8, 2021

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., P15: MS Health Care System/Policy Based Research. Claims Database. Europe and Japan.

Clinical Trials

Practical Cheminformatics

AUGUST 3, 2023

Many papers I’ve read recently use the MoleculeNet dataset, released by the Pande group at Stanford in 2017, as the “standard” benchmark. Clintox – This dataset consists of 1483 SMILES strings and two binary labels indicating whether a molecule is an FDA-approved drug and whether a toxicity outcome has been reported.

Drugs

Agency IQ

SEPTEMBER 15, 2023

Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDA approval so long as it adhered to pre-set terms under the monograph. CHPA supplemented the FDA’s presentation on retail volume with survey results on consumer preferences.

Science

The Pharma Data

SEPTEMBER 10, 2021

“Our research presented at MDS 2021 builds upon our expertise in neuroscience and reinforces our mission to advance the standards of care for people living with these debilitating diseases.” About BOTOX ® BOTOX ® was first approved by the FDA in 1989 for two rare eye muscle disorders – blepharospasm and strabismus in adults.

Doctors

Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. They are used to treat cancers including melanoma and non-small cell lung cancer, but work only for a small fraction of patients.

Research

Drug Target Review

JUNE 21, 2023

The modest sample sizes which are typical in rare disease research make it inherenttly challenging to obtain sufficient power to confidently associate gene variants to phenotypes. 14 In particular in baker’s yeast, researchers could systematically identify gene-gene interactions, including genetic suppressors.

Disease

The Pharma Data

JUNE 18, 2021

Food and Drug Administration (FDA) has approved PREVNAR 20 (Pneumococcal 20-valent Conjugate Vaccine) for the prevention of invasive disease and pneumonia caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes in the vaccine in adults ages 18 years and older. Following today’s FDA approval, the U.S.

Vaccine

The Pharma Data

NOVEMBER 15, 2020

However, doctors are continuing to prescribe the drugs “off-label” for kids with conditions like attention deficit hyperactivity disorder (ADHD), anxiety, depression and conduct disorders, the research found. Food and Drug Administration approval for this off-label usage, according to the study.

FDA Approval

The Pharma Data

AUGUST 25, 2020

“AML is one of the most difficult-to-treat blood cancers, and we’re committed to furthering our research and improving on the standards of care for patients living with this aggressive disease.”. In August 2017, Bristol Myers Squibb received full approval in the U.S. Food and Drug Administration (FDA)-approved test.

Trials

Agency IQ

MAY 17, 2024

The bill is effectively a successor to the RACE for Children Act of 2017, which became law as part of the FDA Reauthorization Act of 2017 (FDARA). However, the FDA then approved another amifampridine product, Ruzurgi, in 2019 for the treatment of LEMS in pediatric patients. DORIS MATSUI (D-Calif.)

FDA

The Pharma Data

AUGUST 22, 2021

a CheckMate -274 primary investigator and Professor of Medicine, Director of Genitourinary Medical Oncology, Co-Director of the Center of Excellence for Bladder Cancer, and Associate Director for Translational Research at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai. Galsky,* M.D., About CheckMate -274.

FDA Approval

The Pharma Data

JANUARY 21, 2021

The 2021 finalists were selected from 1,760 highly qualified entrants, all of whom completed an original research project and extensive application process. Each year, around 2,000 student entrants submit original research in critically important scientific fields of study and are judged by leading experts in their fields.

Science

Agency IQ

JUNE 2, 2023

FDA unveils long-awaited Patient Medication Information proposed rule Since 2017, the FDA has been working on a proposal to create a new type of patient-focused labeling for certain outpatient drug products that would be specifically targeted for patient use.

FDA

The Pharma Data

JUNE 18, 2021

” The pivotal Phase 3 RESONATE-2 study served as the basis for the FDA approval of IMBRUVICA as a single-agent in first-line treatment for CLL/SLL in 2016, following initial approval for relapsed/refractory (R/R) patients in 2014 based on the RESONATE study. 2.2017 were used given 74 percent enrollment in 2017.

FDA Approval

The Pharma Data

MARCH 11, 2021

In 2017, Opdivo was granted accelerated approval by the FDA as a single agent for patients with HCC who have been previously treated with sorafenib. The accelerated approval was based on tumor responses from the Phase 1/2 CheckMate -040 trial.

Treatment

The Pharma Data

JULY 23, 2021

We took this action following the Agency’s industry-wide evaluation of accelerated approvals for checkpoint inhibitors that have not met their post-marketing requirements demonstrating confirmatory benefit. This included a meeting of the Oncologic Drugs Advisory Committee in April and subsequent discussion with the FDA.

Treatment

The Pharma Data

MARCH 16, 2021

Food and Drug Administration (FDA) approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older in August of 2020. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively.

Pharmacokinetics

The Pharma Data

MARCH 16, 2021

Vice President, Rheumatology Disease Area Leader, Janssen Research & Development, LLC. TREMFYA was approved in the U.S. for the treatment of adults with moderate to severe plaque PsO in July 2017 and in July 2020 for adults with active PsA. TREMFYA q4w is not currently FDA-approved.

Treatment