Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

Therapies 52

Therapies Virus DNA Compliance 52

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

Therapies 52

Therapies Research Engineering Clinical Research 52

The Pharma Data

DECEMBER 18, 2020

FDA Approves Orgovyx (relugolix) as the First Oral Gonadotropin-Releasing Hormone (GnRH) Receptor Antagonist for Advanced Prostate Cancer. Food and Drug Administration (FDA) has approved Orgovyx (relugolix) for the treatment of adult patients with advanced prostate cancer. “I BASEL, Switzerland, Dec.

FDA Approval 52

FDA Approval FDA Science Therapies 52

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

Production 134

Production Clinical Trials Trials Therapies 134

The Pharma Data

MAY 4, 2021

INDIANAPOLIS , May 4, 2021 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) is donating COVID-19 therapies to Direct Relief, enabling the humanitarian organization to provide COVID-19 therapies at no cost to low- and lower-middle-income countries most heavily impacted by the pandemic.

Therapies 52

Therapies Hospitals Treatment FDA Approval 52

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Historically, the available drugs and U.S. None offers a cure for PAH.

Clinical Trials 98

Clinical Trials Trials Clinical Research Disease 98

New Drug Approvals

SEPTEMBER 10, 2024

2] Vorasidenib was approved for medical use in the United States in August 2024. [2] 2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2]

FDA Approval 62

FDA Approval FDA Clinical Trials Trials 62

The Pharma Data

NOVEMBER 20, 2020

NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA). TARRYTOWN, N.Y.,

Therapies 52

Therapies FDA Clinical Trials Hospitals 52

The Pharma Data

MAY 4, 2021

Abnormal Laboratory Values: Evaluate at baseline and thereafter according to local patient management practice. Monitor closely when treating patients with abnormal baseline and post-baseline laboratory values. Manage patients according to routine clinical guidelines.

Licensing 40

Licensing Licensing Therapies Laboratories 40

The Pharma Data

AUGUST 17, 2021

Second FDA approved indication for dostarlimab in 2021 GARNET study demonstrated objective response rate of 41.6% This indication received accelerated approval based on tumour response rate and durability of response. Grade 3 or 4 laboratory abnormalities (?2%)

FDA 52

FDA FDA Approval Treatment DNA 52

The Pharma Data

OCTOBER 26, 2020

FDA Actions. FDA Approval: Last week the FDA approved Veklury (remdesivir) for the treatment of COVID-19 requiring hospitalization in adults and pediatric patients (12 years of age and older). Testing Therapies, Antivirals and Vaccines. The drug is an orally active caspase inhibitor. Please read more here.

Vaccine 52

Vaccine FDA Approval Clinical Trials FDA 52

Eye on FDA

APRIL 15, 2020

Bristol Myers Squibb stated in its press release announcing FDA approval of the company’s treatment for multiple sclerosis that the commercial launch would be delayed due to the COVID-19 situation. Why would COVID-19 affect a drug launch? Why would COVID-19 affect a drug launch?

Therapies 55

Therapies Treatment FDA Approval Laboratories 55

The Pharma Data

FEBRUARY 23, 2022

For laboratory workers: TBE vaccination is recommended for those with a potential exposure to the TBE virus (TBEV). Food and Drug Administration (FDA) approved TicoVac™ for active immunization to prevent TBE in individuals 1 year of age and older. 1 TicoVac™ is the only FDA-approved vaccine to help protect U.S.

Vaccine 52

Vaccine FDA Approval Laboratories Virus 52

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. Selman Waksman (middle) in his laboratory, sorting through soil samples with two unidentified assistants.

Vaccine 86

Vaccine Trials Therapies Treatment 86

The Pharma Data

APRIL 8, 2021

“Since the beginning of the pandemic, we have worked to expand the science behind COVID-19 therapies,” said Ilya Yuffa, senior vice president and president of Lilly Bio-Medicines. It is approved and commercially available as OLUMIANT in the U.S. Abnormal Laboratory Values: ?Evaluate Warnings and Precautions ?in

Hospitals 52

Hospitals FDA Approval Laboratories Treatment 52

The Pharma Data

DECEMBER 8, 2020

The majority of this revenue growth is expected to come from the company’s Wave 1 pipeline, which includes 12 unique New Molecular Entities (NMEs), representing potential best-in-class/first-in-class therapies, and its existing 14 global brands. Food and Drug Administration (FDA). ABOUT TAK-721 and TAK-003.

Pharmaceutical Companies 52

Pharmaceutical Companies Therapies FDA Approval Vaccine 52

The Pharma Data

NOVEMBER 16, 2020

In 2020, Regeneron has reinforced our commitment to ‘doing well by doing good,’ as colleagues across the company have worked tirelessly to discover and develop a potential antibody therapy for COVID-19, while at the same time responding to the needs of our colleagues, patients and communities during this public health crisis.”

FDA Approval 52

FDA Approval Science Disease Laboratories 52

The Pharma Data

JANUARY 13, 2021

Drugs developed under the Fast Track program are afforded increased access to FDA staff and may qualify for other programs to further expedite their clinical development, such as priority review and accelerated approval. Brilacidin antiviral research to date has been limited to laboratory-based experiments.

Clinical Trials 52

Clinical Trials Trials Treatment Pharmaceuticals 52

Agency IQ

NOVEMBER 3, 2023

regulatory landscape for probiotics and other microbiome-based therapies Probiotics, a type of microbiome-based therapy containing live micro-organisms, are broadly available on drug store and grocery store shelves, but the regulatory classifications for products containing the same micro-organism can differ dramatically.

Therapies 40

Therapies Science FDA Production 40

The Pharma Data

MARCH 4, 2021

The sBLA is supported by data that include pivotal Phase 3 results evaluating the efficacy and safety of Dupixent in addition to standard-of-care maintenance therapy in children with moderate-to-severe asthma with type 2 inflammation, characterized by elevated blood eosinophil levels and/or raised fractional exhaled nitric oxide (FeNO) levels.

FDA 52

FDA Disease Treatment FDA Approval 52

The Pharma Data

JULY 12, 2021

online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. “Academic labs and pharmaceutical and? Nature Communications, 2021.

Clinical Trials 52

Clinical Trials Drugs Disease Trials 52

The Pharma Data

JUNE 26, 2021

Libtayo is the first immunotherapy to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial, and with this first-in-class approval has the potential to transform treatment for patients in Europe whose cancer has progressed despite HHI treatment ,” said Israel Lowy, M.D.,

Trials 52

Trials Treatment Therapies Disease 52

The Pharma Data

AUGUST 19, 2021

3] While MET inhibitors have recently received accelerated approval in this setting in some regions, the vast majority of patients eventually acquire resistance to these therapies, thus underscoring the need for new treatment options. [4] RYBREVANT TM (amivantamab-vmjw) received accelerated approval by the U.S.

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

NOVEMBER 22, 2020

The FDA approved it under the brand name Gavreto on September 4. The drug is a once-daily oral precision therapy designed for highly potent and selective targeting of oncogenic RET alterations. The drug was approved for that indication on November 13.

FDA 52

FDA Licensing Licensing Treatment 52

The Pharma Data

MARCH 4, 2021

Baricitinib has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of AA. There are currently no FDA-approved treatments for AA. It is approved and commercially available as OLUMIANT in the U.S. associate professor of Dermatology at Yale School of Medicine.

Trials 52

Trials Therapies Treatment Clinical Trials 52

The Pharma Data

MAY 2, 2022

AUSTEDO is the first and only FDA-approved product to treat both tardive dyskinesia in adults and chorea associated with Huntingdon’s disease. If a patient develops akathisia, the AUSTEDO dose should be reduced; some patients may require discontinuation of therapy. Parkinsonism has also been observed with other VMAT2 inhibitors.

Pharmaceuticals 52

Pharmaceuticals Production Treatment Disease 52

The Pharma Data

OCTOBER 22, 2020

With today’s FDA approval, and regulatory approvals or temporary authorizations in approximately 50 additional countries around the world, Veklury® (remdesivir) is one of the tools available today. [ii] Perform hepatic laboratory testing in all patients (see Dosage and administration). Adverse reactions.

Science 52

Science Clinical Trials Hospitals Trials 52

The Pharma Data

MAY 31, 2022

The FDA grants priority review to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. who are unable to control their disease with systemic therapy and are most in need of a treatment option. There are approximately 75,000 people in the U.S.

FDA 52

FDA Disease Trials FDA Approval 52

The Pharma Data

APRIL 4, 2022

FDA granted Breakthrough Therapy designation to Dupixent for the treatment of patients aged 12 years and older with EoE. Priority review is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. In September 2020, the U.S.

FDA 52

FDA Treatment Disease FDA Approval 52

Codon

JUNE 18, 2024

These algae are “one of the most interesting, at least one of the most striking, of the plankton,” wrote Klebahn, who transported the cells back to his laboratory in Hamburg, one hundred kilometers to the south. Fished from Nature At least a dozen laboratories are currently using gas vesicles to study living organisms.

Engineering 65

Engineering Laboratories International Clinical Trials 65

The Pharma Data

AUGUST 3, 2021

Abnormal Laboratory Values: There is limited information regarding use of baricitinib in patients with COVID-19 and any of the following clinical findings: absolute neutrophil count (ANC) <1000 cells/mm 3 , absolute lymphocyte count (ALC) <200 cells/mm 3 , and hemoglobin <8 g/dL. It is approved in the U.S.

Hospitals 52

Hospitals FDA FDA Approval Licensing 52

The Pharma Data

JULY 29, 2021

The most frequently used therapies were corticosteroids (79% of patients, mostly dexamethasone) and remdesivir (19% of patients). Essential safety information on the authorized emergency use of baricitinib includes warnings related to serious infections, thrombosis, abnormal laboratory values, vaccinations and hypersensitivity.

Hospitals 52

Hospitals FDA Laboratories Treatment 52

New Drug Approvals

JANUARY 3, 2023

Adagrasib Formula C32H35ClFN7O2 cas 2326521-71-3 Mol weight 604.1174 Antineoplastic Disease Non-small cell lung cancer 2022/12/12 FDA APPROVED, KRAZATI (Mirati Therapeutics) MRTX-849 MRTX849 KRAS G12C inhibitor MRTX849 Adagrasib , sold under the brand name Krazati , is an anticancer medication used to treat non-small cell lung cancer. [1]

FDA 52

FDA Clinical Trials FDA Approval Treatment 52

The Pharma Data

OCTOBER 29, 2020

“Alopecia areata is a challenging disease that currently has no FDA-approved treatment options, making it difficult for healthcare providers to best serve the needs of these patients,” said Brett King , MD, PhD associate professor of Dermatology at Yale School of Medicine. vice president of immunology development at Lilly.

Therapies 52

Therapies Treatment Disease Clinical Trials 52

The Pharma Data

APRIL 6, 2021

OLUMIANT is a once-daily, oral JAK inhibitor approved in the U.S. FDA-approved labeling for Olumiant includes a Boxed Warning for Serious Infections, Malignancy, and Thrombosis. Test patients for latent TB before initiating Olumiant and during therapy. See the full Prescribing Information here.

Treatment 52

Treatment Clinical Trials Therapies Drugs 52

FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval.

FDA 59

FDA Production FDA Approval Licensing 59

The Pharma Data

DECEMBER 28, 2020

. Moleculin recently announced that the FDA had allowed its request for Investigational New Drug (IND) status for Annamycin, allowing Moleculin to begin a Phase 1B /2 clinical trial in the US for patients with soft tissue sarcoma (STS) that has metastasized to the lungs after first-line therapy for their disease.

FDA 52

FDA Clinical Trials Drugs Pharmaceutical Companies 52