Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. For example, one can fuse a deactivated Cas9 protein to so-called “transcriptional regulators” to either activate or repress genes without directly editing the genome at all.

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The Pharma Data

JULY 2, 2025

FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025.

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The Pharma Data

JUNE 13, 2025

FDA Approves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S. Furthermore, regulators in Europe and Japan are evaluating applications for this new regimen.

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Codon

NOVEMBER 1, 2024

Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease. And finally, earlier this year, Gilead reported an antiretroviral drug, called lenacapavir, that was “100 percent protective” against HIV in a phase 3 trial of 5,300 women in South Africa and Uganda.

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BioPharma Drive: Drug Pricing

APRIL 21, 2025

regulators to clear an oral version of its weight loss drug Wegovy two years after reporting initial Phase 3 results. A spokesperson confirmed the Danish drugmaker has asked U.S.

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BioPharma Drive: Drug Pricing

AUGUST 25, 2023

According to one analyst, the approval was the last hurdle keeping Sandoz’s Tyruko from directly competing in the U.S. market against Biogen’s inflammation-regulating medicine Tysabri.

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Advarra

JULY 26, 2022

with 37 states approving medicinal use and 18 approving recreationally. However, it’s not legal federally and is considered a Schedule I drug by the U.S. Drug Enforcement Administration (DEA), meaning it has no accepted medical use and a high potential for abuse. The FDA is granting INDs for such CBD research.

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Metabolite Tales Blog

APRIL 4, 2023

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. Dermavant’s tapinarof is one such friend.

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The Pharma Data

AUGUST 13, 2020

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. The drug was trialled in two studies of a total of 32 male participants with genetically confirmed DMD.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. Food and Drug Administration (“FDA”) has approved Danyelza (naxitamab-gqgk) 40mg/10ml. This indication is approved under accelerated approval regulation based on overall response rate and duration of response.

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The Pharma Data

DECEMBER 15, 2020

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. Food and Drug Administration (FDA) has approved Klisyri (tirbanibulin) for the topical treatment of actinic keratosis (AK) on the face or scalp. The FDA approval of Klisyri is a significant milestone for Athenex.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov. in the first quarter of 2021.

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The Pharma Data

AUGUST 21, 2020

The US regulator originally approved the drug in 2009 for the treatment of chronic lymphocytic leukaemia as an intravenous infusion with a high dose that was administered by a healthcare provider. The drug is expected to be available in early September and set for a European regulatory review next year. Conor Kavanagh.

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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.

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The Pharma Data

APRIL 19, 2022

Food and Drug Administration (FDA) has approved commercial production at the company’s new CAR T-cell therapy manufacturing facility in Frederick, Maryland. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer. Kite, a Gilead Company (Nasdaq: GILD), today announced the U.S.

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Agency IQ

SEPTEMBER 15, 2023

FDA’s nonprescription advisors find no efficacy for phenylephrine This week, FDA’s Nonprescription Drugs Advisory Committee (NDAC) voted unanimously that current scientific data do not support the efficacy of oral phenylephrine as a nasal decongestant, aligning with FDA analysis — and re-analysis — of data.

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FDA Law Blog: Biosimilars

JUNE 13, 2024

Though some drug companies have been reluctant to delist certain patents from the Orange Book, the District Court of New Jersey just ordered Teva to delist 5 of its patents that it deemed improperly listed. The Court addressed Teva’s valid argument that the Inhaler Patents are drug product patents and thus listable.

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The Pharma Data

JULY 29, 2021

Food and Drug Administration approved the first interchangeable biosimilar insulin product, indicated to improve glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. director of the Office of New Drugs in the FDA’s Center for Drug Evaluation and Research.

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FDA Law Blog: Biosimilars

MARCH 3, 2024

The Act is intended to address national security concerns by prohibiting certain conduct by regulated industry. Effect on FDA approvals— In cases where a biological product or drug needs to change aspects of its manufacturing processes to avoid using a covered equipment or service, will it need to file supplements with FDA for the CMC update?

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Pharmaceutical Development Group

DECEMBER 19, 2021

Start Up and Generic Pharmaceutical Drug and Biologic Companies have high quality, affordable products that improve the quality of life for their patients. In a FDA Pre-Submission that leads to FDA Approval, more does not equal better and more does not equal relevance to a specific Pharmaceutical Drug or Biologic Product.

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Pharmaceutical Development Group

JANUARY 7, 2022

Start Up and Generic Pharmaceutical Drug and Biologic Companies have high quality, affordable products and biosimilars that improve the quality of life for their patients. Seamless application of Data Standards, Data Integrity, begins no later than DMPK Protocols and all other Data filed in the Drug History File and used in the CMC.

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KIF1A

JULY 1, 2023

This activity may be regulated by the K-loop, which is known to be important for KIF1A’s long-range movement. But drug R&D is an uncertain process, and many therapeutics get shelved during preclinical or clinical research, before they ever reach a patient.

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The Pharma Data

MAY 17, 2021

The authority to change drug labels outside of considerations for new safety information “could encourage third parties, such as academic investigators, insurance companies, and cooperative trial groups, to initiate such changes,” they wrote. .

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DrugBank

APRIL 24, 2025

Epigenetics , the study of changes in gene activity that occur without altering the DNA sequence, has revolutionized our understanding of gene expression regulation. Epigenetic drugs, which target the enzymes and processes involved in these modifications, represent a novel approach to precision medicine.

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FDA Law Blog: Biosimilars

JANUARY 4, 2024

The American Conference Institute (“ACI”) will be hosting the go-to forum for critical updates on OTC regulation and enforcement, monograph reform, ACNU and advertising essentials… and FDA Law Blog readers can get a discount. Deb along with fellow panelists Kyle Y.

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FDA Law Blog: Biosimilars

SEPTEMBER 19, 2023

The six-page statement explains that “Brand drug manufacturers may be harming generic competition through the improper listing of patents in the. Of course, the statute says that only drug formulation, composition, or method of use patents are listable, but FDA has not defined the scope of the “drug” that must be covered by the patent.

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Drug Target Review

SEPTEMBER 19, 2023

The US FDA Modernisation Act 2.0., puts an end to the previous mandate that all drugs need to be tested on animals prior to human clinical trials. 3-5 Boston biotech, Emulate, established liver-on-chip device is designed to accurately measure liver toxicity and predict drug induced liver injury (DILI).

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FDA Law Blog: Biosimilars

AUGUST 8, 2024

With apologies to Curtis Mayfield) By the close of the public comment period for the Drug Enforcement Administration’s (“DEA’s”) proposal to reschedule marijuana two weeks ago, the agency had received over 43,500 comments. Some states have authorities that regulate marijuana exclusively. Department of Justice (“DOJ”) and DEA respond?

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FDA Law Blog: Biosimilars

NOVEMBER 5, 2023

Dr. Won will share his expertise and insights on the intricacies of the United States Food and Drug Administration (FDA) regulations pertaining to medical devices, with a special focus on class II and class III hearing devices. The comprehensive agenda for the symposium can be accessed in its entirety here.

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FDA Law Blog: Biosimilars

OCTOBER 1, 2024

Or consider Vanda’s challenge that FDA’s approval of a generic version of one of the company’s drugs violated the Appointments Clause of the Constitution (U.S. 2) because the FDA employees who approved the application were not “Officers of the United States.” II, § 2, cl. VANDA PHARMACEUTICALS, INC. 23-5200 (D.C.

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FDA Law Blog: Biosimilars

AUGUST 12, 2024

In the veterinary world, where rolling applications are common, the testing phase is usually particularly important because the review phase, which starts only when the last component of the rolling New Animal Drug Application (NADA)—called the Administrative NADA—is submitted, is short, typically around 60 days.

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The Pharma Data

MAY 4, 2021

Pfizer plans to file for full FDA approval of Covid vaccine at the end of this month ( CNBC ). The FDA is set to authorize the Pfizer-BioNTech vaccine for those 12-15 years old by early next week. House Democrats ramp up pressure on drug prices ( Politico ). In Focus: US. NYTimes ).

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The Pharma Data

MAY 27, 2022

billion in Medicare funds in 2019 on drugs whose clinical benefits have yet to be confirmed by the Food and Drug Administration, a new study led by researchers from the Johns Hopkins Bloomberg School of Public Health suggests. The program provides drug approval based on what is known as a surrogate endpoint.

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Drug Target Review

JULY 5, 2024

1 Considerations for creating complex organoids Despite the numerous advantages of using brain organoids in drug discovery, some limitations should be considered. 1 Considerations for creating complex organoids Despite the numerous advantages of using brain organoids in drug discovery, some limitations should be considered.

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FDA Law Blog: Biosimilars

MARCH 12, 2024

Koblitz — After years of silence from FDA on whether certain patents could be listed in the Orange Book, some manufacturers of drug and device combination products have had a rude awakening lately. In fact, federal law and regulation appear to require AbbVie to list these patents.”

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PPD

AUGUST 5, 2024

Historically, the available drugs and U.S. 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.

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The Pharma Data

MAY 7, 2021

regulators to seek approval of our COVID-19 vaccine based on our pivotal Phase 3 trial and follow-up data.”. The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. “Following the successful delivery of more than 170 million doses to the U.S. CEO and Co-founder of BioNTech. “We

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