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

FEBRUARY 24, 2022

Jardiance is also approved to reduce the risk of cardiovascular death in adults with type 2 diabetes and established cardiovascular disease, and to reduce the risk of death and hospitalization in patients with heart failure and low ejection fraction.

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The Pharma Data

JUNE 26, 2021

Food and Drug Administration approved Pradaxa (dabigatran etexilate) oral pellets to treat children 3 months to less than 12 years old with venous thromboembolism (a condition where blood clots form in the veins) directly after they have been treated with a blood thinner given by injection for at least five days.

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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

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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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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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. BASEL, Switzerland, Dec.

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The Pharma Data

JUNE 26, 2023

FDA approves Pfizer’s LITFULO™ (ritlecitinib) for adults and adolescents with severe alopecia areata Pfizer Inc. Food and Drug Administration (FDA) has approved LITFULO™ (ritlecitinib), a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata. with placebo.

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The Pharma Data

JULY 12, 2021

Food and Drug Administration (FDA) has approved finerenone, the first non-steroidal, selective mineralocorticoid receptor (MR) antagonist, under the brand name Kerendia ®. Despite guideline-directed therapies, patients with CKD and T2D remain at high risk of CKD progression and cardiovascular events.

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Eye on FDA

AUGUST 4, 2021

Each committee is comprised of a number of experts, diverse for their expertise, geography and specialty – with some focused on statistics while others may be focused on areas of concentration within the category, such as the treatment of pediatric patients.

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The Pharma Data

MAY 5, 2021

A total of 197 patients in the study arm had at least one of the composite endpoint events, compared with 312 patients in the placebo arm. The approval was based on results in a prespecified interim analysis of the first 264 patients of the ongoing KEYNOTE-811 trial.

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The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has approved Repatha® (evolocumab) as an adjunct to diet and other LDL cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous hypercholesterolemia (HeFH) to scale back LDL-C. No new safety risks were identified.5

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The Pharma Data

JUNE 22, 2023

FDA approval Pfizer (NYSE: PFE) announced that the U.S. Metastatic CRPC is a form of prostate cancer that has spread beyond the prostate gland and has progressed despite medical or surgical treatment to lower testosterone. Therefore, new first-line treatment options are needed to reduce the risk of disease progression or death.

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The Pharma Data

DECEMBER 16, 2020

We are entering an area with significant unmet medical need since the current treatment paradigm for GBM remains bleak, as this aggressive and currently incurable form of brain cancer continues to claim high mortality rates. These statements relate to future events, future expectations, plans and prospects.

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The Pharma Data

OCTOBER 14, 2020

Food and Drug Administration (FDA) has approved WAKIX® (pitolisant) for the treatment of cataplexy in adult patients with narcolepsy. WAKIX received FDA approval for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019. Drug Enforcement Administration.

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The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. 1 Secondary endpoints included time to cytotoxic chemotherapy, time to pain progression, time to chronic opioid use, and time to skeletal-related events. [3] 10 months. [5]

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The Pharma Data

DECEMBER 21, 2020

We know from our research that the majority of men prefer an oral treatment to an injection,” Myovant CEO Lynn Seely said. The secondary endpoint for increasing lifespan for patients with metastatic prostate cancer came in at 74% still alive after treatment with Orgovyx compared to 75% of those on Lupron. .

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The Pharma Data

AUGUST 19, 2021

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].

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The Pharma Data

APRIL 28, 2023

Teva and MedinCell Announce FDA Approval of UZEDY™ (risperidone) Extended-Release Injectable Suspension, a Long-Acting Subcutaneous Atypical Antipsychotic Injection, for the Treatment of Schizophrenia in Adults Teva Pharmaceuticals, a U.S. The initiation of treatment requires no loading dose or oral supplementation.

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The Pharma Data

MARCH 26, 2021

Despite continuous innovations in the treatment landscape, unmet needs remain. If approved by the European Commission, ponesimod has the potential to help more people living with relapsing forms of MS.”. 5] The MAA will now be reviewed by the European Commission (EC) for the treatment of adults with RMS. vs. 10.4%).

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The Pharma Data

FEBRUARY 25, 2022

If approved, rimegepant will be the first oral CGRP receptor antagonist in the EU, and the only migraine medication approved for both acute and preventive treatment. In these studies, rimegepant was safe and well-tolerated with rates of adverse events similar to that of placebo.

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The Pharma Data

JANUARY 17, 2021

a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, today announced dose administration for the first patient in a Phase 2a clinical study of the company’s lead drug candidate, STP705, for the treatment of cutaneous basal cell carcinoma. GAITHERSBURG, Md.

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The Pharma Data

JULY 8, 2021

ADUHELM is indicated for the treatment of Alzheimer’s disease. Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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The Pharma Data

APRIL 6, 2021

Food and Drug Administration (FDA) has extended the review period for the supplemental New Drug Application (sNDA) for baricitinib for the treatment of adults with moderate to severe atopic dermatitis (AD). OLUMIANT is a once-daily, oral JAK inhibitor approved in the U.S.

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The Pharma Data

MAY 31, 2022

The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment.

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The Pharma Data

MARCH 18, 2022

Opdualag is a first-in-class, fixed-dose dual immunotherapy combination treatment of the PD-1 inhibitor nivolumab and novel LAG-3-blocking antibody relatlimab 1. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma.1

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The Pharma Data

FEBRUARY 22, 2022

Food and Drug Administration (FDA) for the adjunctive treatment of major depressive disorder (MDD) in patients who are receiving ongoing antidepressant therapy. In both of these studies, safety data were consistent with the established safety profile of cariprazine across indications, with no new safety events identified.

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The Pharma Data

APRIL 26, 2022

This approval follows the recent sNDA approval for Veklury for the treatment of non-hospitalized adult and adolescent patients who are at high risk of progression to severe COVID-19. For hospitalized pediatric patients who do not require invasive mechanical ventilation and/or ECMO, a 5-day treatment course is recommended.

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The Pharma Data

MAY 6, 2022

The adverse event profile was generally consistent with other clinical safety data for the vaccine, with a favorable safety profile observed in individuals with at least 6 months of safety follow-up after the second dose.

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The Pharma Data

MARCH 30, 2021

2] – The atogepant application demonstrates AbbVie’s longstanding commitment to providing multiple migraine treatment options, including BOTOX® (onabotulinumtoxinA), a preventive treatment for those with chronic migraine, and UBRELVY® (ubrogepant), an acute treatment for adults with migraine. .

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The Pharma Data

JANUARY 13, 2021

Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma. Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform. “I About the Phase 1/2 Trial for HPN217.

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The Pharma Data

JUNE 28, 2021

1,2 The most commonly reported adverse events in patients treated with RINVOQ were acne, nasopharyngitis and upper respiratory tract infections. “Despite available treatments, many people with moderate to severe forms of this disease continue to experience a relentless and burdensome cycle of skin and itch symptoms. .

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The Pharma Data

NOVEMBER 8, 2021

“The positive outcomes strengthen the efficacy and safety profile of erenumab as a migraine prevention treatment for patients with migraine.” “The positive outcomes strengthen the efficacy and safety profile of erenumab as a migraine prevention treatment for patients with migraine.” versus 38.9%). .

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DECEMBER 1, 2020

In order to listen to the audio webcast, interested parties can register and access the live webcast under “News/Events” through the “Investors” section of the Aurinia corporate website at www.auriniapharma.com. The Company is currently seeking FDA approval of voclosporin for the potential treatment of LN.

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The Pharma Data

FEBRUARY 13, 2022

Food and Drug Administration issued an emergency use authorization (EUA) for a new monoclonal antibody for the treatment of COVID-19 that retains activity against the omicron variant. Treatment with bebtelovimab has not been studied in patients hospitalized due to COVID-19. The issuance of an EUA is different than an FDA approval.

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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. As a result, researchers now often use composite endpoints in lieu of the customary single primary endpoint.

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The Pharma Data

JANUARY 26, 2021

Across 1,035 patients, there were 11 events (2.1 percent) in patients taking therapy and 36 events (7.0 These data further support our belief that bamlanivimab and etesevimab together have the potential to be an important treatment that significantly reduces hospitalizations and death in high-risk COVID-19 patients.

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