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

SEPTEMBER 7, 2020

While the approval has been awarded, the FDA warned that continued approval may be dependent on Genentech reinforcing these positive data with a future confirmatory clinical trial. These benefits were seen in patients regardless of RET fusion partner or central nervous system involvement. Matt Fellows. Source link.

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The Pharma Data

AUGUST 10, 2020

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy available for the rare hereditary genetic condition in US and the second overall to be approved for the disease.

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The Pharma Data

OCTOBER 29, 2021

Novartis blazoned moment that the US Food and Drug Administration (FDA) approved Scemblix ® (asciminib) for the treatment of habitual myeloid leukemia (CML) in two distinct suggestions. In cases with Ph CML-CP who had endured resistance or dogmatism to at least two TKIs, the ASCEMBL trial showed that1-3.Scemblix

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The Pharma Data

AUGUST 27, 2020

Roche’s Executive Vice President, Chief Medical Officer, Head of Global Product Development and Co-Founder of Foundation Medicine Inc. The test is FDA-approved to report short variants in 311 genes including rearrangements and copy number losses in BRCA1 and BRCA2 genes.

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The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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The Pharma Data

JULY 8, 2021

The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language). Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has approved TICOVAC (tick-borne encephalitis (TBE) vaccine) 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. Following today’s FDA approval, the U.S. in 1-15 year olds and 98.7-100%

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FDA Law Blog: Drug Discovery

OCTOBER 11, 2023

Snow — On September 18, 2023, FDA published an updated, final iteration of guidance for immediate implementation entitled, “ Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies.” hurricane) or public health emergency (e.g.,

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Drug Target Review

JANUARY 8, 2024

With cellular rejuvenation, our goal is to improve healthspan so as to maximise the number of healthy and productive years one has in their lifetime, not just their total number of years. Jerry began his career at Merck and was extensively involved in multiple blockbuster product launches. a commercial stage pharmaceutical company.

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Codon

JULY 14, 2024

receives a blood transfusion or a blood product every two seconds. But currently, traditional blood transfusions face significant challenges, including a dwindling pool of donors and a limited shelf life for stored blood products. However, the subsequent death of another patient thrust Denys into a contentious trial.

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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. Klisyri is the first FDA approved branded proprietary product for Athenex and will be launched in partnership with Almirall in the U.S. The FDA approval of Klisyri is a significant milestone for Athenex.

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The Pharma Data

OCTOBER 30, 2020

To increase demand for products during the novel coronavirus pandemic, the US Food and Drug Administration (FDA) has already approved at-home testing and investigated the effectiveness of malaria pills and other antiviral drugs to treat Covid-19 9. FDA approves first treatment for Covid-19.

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The Pharma Data

JANUARY 11, 2021

based subsidiary of Terumo and a global neurovascular company, announced today the FDA Approval of the PMA Supplement for the WEB 17 System, a new addition to the WEB Aneurysm Embolization System for the treatment of intracranial wide neck bifurcation aneurysms. . ALISO VIEJO, Calif. , www.terumo.com.

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The Pharma Data

MAY 27, 2022

Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

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The Pharma Data

DECEMBER 17, 2020

Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy. Lupus causes the production of proteins called autoantibodies in the immune system, which attack tissues and organs, including the kidneys.

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The Pharma Data

NOVEMBER 2, 2020

FDA Approves Bronchitol (mannitol) Inhalation Powder to Improve Pulmonary Function in Adult Patients with Cystic Fibrosis. Food and Drug Administration (FDA) approval of Bronchitol (mannitol) inhalation powder. Food and Drug Administration (FDA) approval of Bronchitol (mannitol) inhalation powder.

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The Pharma Data

MAY 5, 2021

Its indicated dose of 8 mg is notably higher than the previously approved doses of 2 and 4 mg for other naloxone nasal spray products. . The approval was based on results in a prespecified interim analysis of the first 264 patients of the ongoing KEYNOTE-811 trial. Patients in the multicenter, double?blind,

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The Pharma Data

JANUARY 14, 2021

Food and Drug Administration (FDA) approved Pfizer ’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. Manuel Esteban/Shutterstock.

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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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The Pharma Data

FEBRUARY 24, 2022

– FDA approval marks a significant breakthrough for the approximately 3 million adults in the U.S. “In its phase III trials, empagliflozin has shown a statistically significant and clinically meaningful benefit in patients with heart failure across the spectrum of ejection fraction,” said Javed Butler, M.D.,

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The Pharma Data

NOVEMBER 12, 2021

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. Today, the U.S.

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The ChEMBL-og

APRIL 16, 2024

A significant effort has been made to correctly map the drug form of the EMA data by manually inspecting different EMA sources of information, such as the Product Information (Annex I: Summary of Product Characteristics and Annex III: Labelling and Package Leaflet) and/or Assessment Report, where available. University of Dundee: T.

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Advarra

JUNE 20, 2023

of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. years , with biotechnology-derived products potentially adding another year or more. Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community.

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The Pharma Data

JANUARY 15, 2021

Food and Drug Administration (FDA) approval of Darzalex Faspro ® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1]

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The Pharma Data

AUGUST 7, 2021

Nexviazyme has been shown in clinical trials to provide patients with improvements in respiratory function and walking distance. In the pivotal Phase 3 trial (COMET), Nexviazyme showed improvements in respiratory function and walking distance measures in people with LOPD and established its safety profile. meters farther (SE=9.9)

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The Pharma Data

DECEMBER 3, 2020

Food and Drug Administration (FDA) has approved ORLADEYO (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and young patients 12 years and older. The approval was based on data from the Phase 3 APeX-2 trial, which showed that ORLADEYO could significantly reduce attacks at the 24-week mark.

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The Pharma Data

SEPTEMBER 6, 2020

Basel, 7 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the United States (US) Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test.

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The Pharma Data

DECEMBER 21, 2020

The Phase III study was conducted as an open-label trial with patients receiving either an Orgovyx pill daily or injections of Abbvie’s Lupron as an injection every three months. Because prostate cancer is fueled by testosterone production, both drugs decrease levels of the hormone in the body. .

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The Pharma Data

NOVEMBER 16, 2020

November 16, 2020 — An independent data and safety monitoring board (DSMB) overseeing the Phase 3 trial of the investigational COVID-19 vaccine known as mRNA-1273 reviewed trial data and shared its interim analysis with the trial oversight group on Nov. 37% of trial volunteers are from racial and ethnic minorities.

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The Pharma Data

FEBRUARY 24, 2022

For today’s approval, Jardiance’s safety and effectiveness were evaluated as an adjunct to standard of care therapy in a randomized, double-blind, international trial comparing 2,997 participants who received Jardiance, 10 mg, once daily to 2,991 participants who received the placebo. Source link: [link].

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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. Orgovyx (relugolix) FDA Approval History.

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The Pharma Data

MAY 6, 2022

Food and Drug Administration (FDA) to expand the approval of COMIRNATY® (COVID-19 Vaccine, mRNA) to include individuals ages 12 through 15 years. The sBLA includes updated longer-term follow-up data from the companies pivotal Phase 3 clinical trial of 2,228 participants 12 through 15 years of age.

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The Pharma Data

DECEMBER 16, 2020

The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy. The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy.

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Advarra

NOVEMBER 17, 2022

In 2016, the Food and Drug Administration (FDA) approved Spinraza (nusinersen). While the FDA’s approval of nusinersen may not seem extraordinary, it was. Nusinersen’s approval marked the first time nonclinical data supported conducting initial clinical trials involving children. Why This Guidance Now?

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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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DrugBank

MAY 6, 2024

A Wave of Innovations The year 2024 has already proven to be significant for the field of biologics, witnessing a notable increase in FDA approvals. In the first quarter alone, the FDA approved 9 new biologics, a substantial rise from the 5 approvals during the same period in 2023.

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