Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Such off-target cuts are obviously a big concern when developing human therapies, because they can cause mutations that lead to cancer or other diseases.

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The Pharma Data

JULY 2, 2025

Importantly, the treatment effect emerged early in the course of therapy and continued to increase over time. Despite existing therapies, many PAH patients remain at substantial risk. Patients were randomized 1:1 to receive either WINREVAIR or placebo, both in combination with maximum-tolerated background therapy. Source link

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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. Subsequently, KEYTRUDA is used as monotherapy following the completion of adjuvant therapy.

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Codon

NOVEMBER 1, 2024

Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease. It demands protocols, regulations, and collaborative efforts between human beings. It involves pragmatism and cultural sensitivity.

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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

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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The Pharma Data

SEPTEMBER 7, 2020

Genentech’s once-daily oral therapy Gavreto (pralsetinib) has secured FDA backing in the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), it has emerged. In 27 treatment-naïve NSCLC patients, the ORR was found to be 70% and CRR was 11%.

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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. If this benefit cannot be proven, the FDA may withdraw its authorisation. . of normal at baseline to 5.9%

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. The FDA granted approval under the accelerated approval regulation. NEW YORK, Nov.

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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. NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the U.S. BUFFALO, N.Y.,

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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. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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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.

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The Pharma Data

AUGUST 21, 2020

Kesimpta targets and delivers B-cell therapy and has shown to have superior efficacy and safety profile compared to similar treatments. 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.

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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.

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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. These therapies aim to reverse abnormal epigenetic patterns, restore normal gene function, and offer hope for previously uncurable diseases.

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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. Big three drug distributors blame doctors, regulators in trial over opioid epidemic ( Reuters ).

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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.”. Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to the Pfizer-BioNTech COVID-19 Vaccine. CEO and Co-founder of BioNTech. “We

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The Pharma Data

SEPTEMBER 7, 2020

The personalised cancer treatment has received conditional approval in the US. US regulators have approved Genentech’s Gavreto (pralsetinib) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC).

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The Pharma Data

MAY 17, 2021

. In a Viewpoint, Bishal Gyawali, MD, PhD, and colleagues at the Program on Regulation, Therapeutics and Law (PORTAL) research group at Harvard Medical School and Brigham & Women’s Hospital suggested improving FDA’s authority to modify drug package insert for situations such as drug repurposing and de-escalation of therapy.

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The Pharma Data

APRIL 14, 2021

Food and Drug Administration (FDA) has approved the company’s supplemental Biologics License Application for Xolair® (omalizumab) prefilled syringe for self-injection across all approved U.S. with Xolair since its initial approval in 2003. indications. Approximately 460,000 patients have been treated in the U.S.

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FDA Law Blog: Biosimilars

FEBRUARY 27, 2024

156, a patent may be extended only once (even if it would be eligible for extension on more than one occasion because it applies to several FDA-approved products), and only one patent may be extended for each regulatory review period. PTE applications were submitted to FDA for each of U.S. To that end, 35 U.S.C. § 156(c)(4)

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The Pharma Data

SEPTEMBER 7, 2020

Novartis will present 48 abstracts from its leading MS portfolio, including new data on recently FDA-approved Kesimpta ® (ofatumumab) — the first and only self-administered, targeted B-cell therapy for relapsing forms of MS (RMS )— Mayzent ® (siponimod) and Gilenya ® (fingolimod) .

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NIH Director's Blog: Drug Development

JULY 20, 2017

Years of hard work, supported by the National Institutes of Health and the Cystic Fibrosis Foundation, painstakingly worked out the normal function of the protein that is altered in CF, called the cystic fibrosis transmembrane regulator (CFTR). That combination therapy was designed to help CF patients with two copies of the F508del mutation.

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Drug Target Review

OCTOBER 17, 2024

It is not known whether CD24 on cancer cells has a unique epitope that can be specifically targeted for cancer therapy. By definition, the do-not-eat-me signal refers to phagocytosis of tumour cells by macrophages, although we and others have shown that the CD24/Siglec-10 pathway also regulates the function of T cells and NK cells.

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The Pharma Data

MARCH 11, 2021

Retevmo was approved under the FDA’s Accelerated Approval regulations based on the LIBRETTO-001 Phase 1/2 trial’s endpoints of overall response rate (ORR) and duration of response (DoR). Session Title: Targeted Therapy and Ovarian Cancer Trials. Abstract Number: CT011. Retevmo is an U.S.

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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. There are currently no approved therapies to treat this disorder’s hyperphagia, anxiety, or metabolic aspects.

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The Premier Consulting Blog

OCTOBER 11, 2022

The treatment had been granted breakthrough therapy designation, but FDA approval would ultimately rest on the sponsor’s ability to demonstrate clinically meaningful improvement. Key Takeaway RWD and RWE play an important role in rare diseases, where capturing relevant data to support drug development is challenging.

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Vial

DECEMBER 5, 2023

In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Fountzilas et al.

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Advarra

JANUARY 23, 2024

limited efficacy of experimental therapy) Change to the research resulting in increased burden or discomfort New alternative therapy availability (e.g., FDA approval of a new drug for the condition under study) Impact of participation on alternative therapies (e.g.,

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New Drug Approvals

OCTOBER 24, 2023

Molecular Weight: 631.700 FDA APPROVED, To treat moderately to severely active ulcerative colitis in adults, 10/12/2023 Velsipity Etrasimod , sold under the brand name Velsipity , is a medication that is used for the treatment of ulcerative colitis (UC). [1] “FDA Approves New Drug for Ulcerative Colitis” Medscape.

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FDA Law Blog: Biosimilars

JULY 24, 2023

Approximately six weeks after FDA approved Lumryz and issued its clinical superiority decision , Jazz filed a Complaint against FDA in the District Court of D.C. Jazz also argues that, notwithstanding the language that instructs FDA to promulgate regulations implementing the clinical superiority provisions at 21 U.S.C.

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The Pharma Data

JANUARY 29, 2021

AstraZeneca continues to work with regulatory authorities around the world to support their ongoing rolling reviews for emergency supply or conditional approval during the health crisis. AZD1222 (SARS-CoV-2 vaccine) FDA Approval History. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca. Source link.

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The Pharma Data

JANUARY 29, 2021

AstraZeneca continues to work with regulatory authorities around the world to support their ongoing rolling reviews for emergency supply or conditional approval during the health crisis. AZD1222 (SARS-CoV-2 vaccine) FDA Approval History. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca. Source link.

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FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

156, as added by the 1984 Hatch-Waxman Amendments, for certain FDA-regulated products. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. Karst — Earlier this week, we posted Part 1 of our three-part series on U.S. 6,953,787 [] for [PTE] request under 35 U.S.C. §

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The Pharma Data

DECEMBER 30, 2020

The decision to approve the vaccine was taken under Regulation 174 of the Human Medicine Regulations 2012, which enables rapid emergency regulatory approvals to address significant public health issues such as a pandemic. AZD1222 (SARS-CoV-2 vaccine) FDA Approval History. Source: AstraZeneca. Source link.

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New Drug Approvals

DECEMBER 16, 2023

1 Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase. 2 This is of particular importance to PNH, where one of the disease hallmarks is the mutation of the PIGA gene. . twitter +919321316780 call whatsaapp EMAIL. 5 December 2023. Click here to purchase.

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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).

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