Pharmaceutical Technology

MARCH 1, 2023

Kevzara has been approved as the first and only biologic indicated for PMR adult patients who did not respond to corticosteroids or who cannot tolerate corticosteroid taper. Developed using VelocImmune technology of Regeneron, Kevzara binds to the IL-6 receptor specifically and inhibits IL-6-mediated signalling.

FDA Approval 245

FDA Approval Clinical Trials Clinical Trials Trials 245

Pharmaceutical Technology

MARCH 10, 2023

It is a third-generation, selective and structurally unique, small molecule CGRP receptor antagonist and is currently under clinical development with oral and intranasal formulations. The company stated that the regulatory approval expands its migraine portfolio, which includes oral therapy for acute and preventive treatment.

FDA Approval 246

FDA Approval Pharmacy Trials Clinical Development 246

Pharmaceutical Technology

JANUARY 9, 2023

Leqembi is indicated to treat mild cognitive impairment or mild dementia stage of the disease in patients whose treatment started in clinical trials. The regulatory approval is based on the data obtained from the Phase II trial. Leqembi met the primary endpoint and all the critical secondary endpoints in the Clarity AD trial.

FDA Approval 246

FDA Approval Antibody Clinical Trials Clinical Trials 246

Pharmaceutical Technology

APRIL 27, 2023

Seres president and CEO Eric Shaff said: “Seres has led the development of microbiome therapeutics, and today’s FDA approval of Vowst as the first orally administered microbiota-based therapeutic for the prevention of recurrent C difficile infection marks a tremendous milestone for the patient community, and for Seres. “We

FDA Approval 246

FDA Approval Regulation Trials Development 246

XTalks

APRIL 15, 2025

Healthy, nonpregnant women with no structural or functional urinary tract abnormalities typically develop uUTIs, which E.coli usually causes. The FDA indicates Blujepa specifically to treat uUTIs caused by E.coli and Staphylococcus saprophyticus. However, the field has seen some recent activity with several notable FDA approvals.

FDA Approval 66

FDA Approval Bacteria DNA Bacterium 66

Pharmaceutical Technology

JUNE 9, 2023

Biopharmaceutical company Novaliq has received approval from the US Food and Drug Administration (FDA) for VEVYE (cyclosporine ophthalmic solution) 0.1% VEVYE, the development name of which is CyclASol, is a cyclosporine, solubilised solution in a new, water-free excipient. to treat the signs and symptoms of dry eye disease.

FDA Approval 246

FDA Approval Bioavailability Containment Drugs 246

XTalks

NOVEMBER 7, 2024

Emrosi’s approval was based on data from a pair of Phase III clinical trials for the treatment of rosacea. The trials met all co-primary and secondary endpoints, with participants having successfully completed the 16-week treatment with no significant safety issues.

FDA Approval 105

FDA Approval Dermatology Bacteria Clinical Trials 105

Pharmaceutical Technology

SEPTEMBER 19, 2022

bluebird bio was granted a rare paediatric priority review voucher following approval. The latest development is based on findings from the single-arm, open-label Phase II/III ALD-102 (Starbeam) and Phase III ALD-104 clinical trials. thalassemia.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

XTalks

NOVEMBER 15, 2024

Syros Pharmaceuticals announced that its cancer drug tamibarotene failed to meet its primary endpoint in the SELECT-MDS-1 Phase III trial. The placebo-controlled SELECT-MDS-1 trial involved 190 patients with complete response rate (CRR) being the study’s primary endpoint. percent and 18.8

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Trials HR Immune Response FDA Approval 105

Pharmaceutical Technology

APRIL 27, 2023

Vertex Pharmaceuticals chief medical officer and global medicines development and medical affairs executive vice-president Carmen Bozic stated: “Since its initial approval, Trikafta has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis. “We

FDA Approval 246

FDA Approval In-Vitro Drugs Gene 246

Pharmaceutical Technology

FEBRUARY 3, 2023

The US Food and Drug Administration (FDA) has granted approval to Amgen and AstraZeneca ’s Tezspire (tezepelumab-ekko) for self-administration using a single-use, pre-filled pen for severe asthma patients aged 12 years and above. Asthma control improvement and safety profile were consistent with previous clinical trials.

FDA Approval 147

FDA Approval Antibody Clinical Trials Clinical Trials 147

Pharmaceutical Technology

JANUARY 30, 2023

Eli Lilly and Company ’s (Lilly) oncology unit Loxo@Lilly has secured the US Food and Drug Administration (FDA) approval for Jaypirca (pirtobrutinib) to treat relapsed or refractory mantle cell lymphoma (MCL). Lilly stated that the continued approval of Jaypirca for this indication is based on clinical benefit in a confirmatory trial.

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FDA Approval Trials Drugs Development 147

XTalks

FEBRUARY 25, 2025

Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDA approval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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Gene Therapy Gene Trials Clinical Trials 264

XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Date of FDA approval : March 29, 2024 Approved for : To detect exposure to human parvovirus B19. TriClip size comparison photo. Photo courtesy of Abbott Medical.

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FDA Approval Manufacturing Development RNA 105

Pharmaceutical Technology

MAY 1, 2023

Teva Pharmaceuticals and MedinCell have received approval from the US Food and Drug Administration (FDA) for Uzedy (risperidone) extended-release injectable suspension to treat adult patients with schizophrenia, a chronic, progressive, and severely debilitating mental health disorder.

FDA Approval 189

FDA Approval Clinical Trials Clinical Trials Trials 189

World of DTC Marketing

AUGUST 27, 2021

SUMMARY: Over half of all clinical trial sites are outside the U.S. 87 % of all subjects in recent biologics trials were enrolled outside the U.S. DTC may or may not work for clinical trial enrollment it really depends on the condition and risk as judged by the patient. 78 % of all subjects were enrolled outside the U.S.

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Clinical Trials Clinical Trials Trials Drugs 187

Pharmaceutical Technology

FEBRUARY 9, 2023

The US Food and Drug Administration (FDA) has granted approval for Regeneron Pharmaceuticals’ Eylea (aflibercept) injection to treat the retinopathy of prematurity (ROP) in preterm infants. A VEGF inhibitor, Eylea is jointly developed by Bayer and Regeneron Pharmaceuticals.

FDA Approval 130

FDA Approval Trials Development Drugs 130

Pharmaceutical Technology

FEBRUARY 1, 2023

The US Food and Drug Administration (FDA) has granted approval for Ellipses Pharma’s investigational new drug (IND) application for EP0042 to treat acute myeloid leukaemia (AML). The dual FLT-3 and Aurora kinase inhibitor EP0042 is being developed to treat AML patients who have developed FLT3 inhibitor resistance.

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FDA Approval In-Vivo In-Vitro Trials 130

Pharma Mirror

MARCH 10, 2021

A post-pandemic pharma report from CPhI predicts the next few years will deliver strong growth in pharma innovation, with oncology and rare diseases potentially the biggest beneficiaries of positive covid legacies – including accelerated development timelines, remote trials and new technologies in genetic and target screening.

FDA Approval 130

FDA Approval Genetics Trials Development 130

XTalks

JANUARY 2, 2025

The IPO proceeds aim to primarily support the ongoing development of Anteris DurAVR transcatheter heart valve (THV) system, which aims to improve treatment outcomes for aortic stenosis. Trading began on the Nasdaq Global Market on December 13, 2024, under the ticker symbol “AVR,” with the offering closed on December 16, 2024.

FDA Approval 110

FDA Approval Marketing Clinical Trials Clinical Trials 110

FDA Law Blog

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Production Clinical Development Development Regulation 69

Pharmaceutical Technology

SEPTEMBER 5, 2022

Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.

FDA Approval 130

FDA Approval Genotype Clinical Trials Clinical Trials 130

Pharmaceutical Technology

FEBRUARY 17, 2023

The agency proposes that pharma move away from the common maximum tolerated dose (MTD) to trial designs that prioritise drug dosage optimisation at different stages. Since this approval, KRAS inhibitors have risen in popularity with the FDA also approving Mirati Therapeutics’ (NASDAQ:MRTX) inhibitor Krazati (adagrasib) as a therapy for NSCLC.

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Clinical Trials Clinical Trials Trials Drugs 289

Pharmaceutical Technology

JUNE 14, 2023

Ipsen has received US Food and Drug Administration (FDA) approval for Bylvay (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome (ALGS).

FDA Approval 130

FDA Approval Genetics Trials Medicine 130

Pharmaceutical Technology

JANUARY 12, 2023

Airsupra has been approved for the as-needed treatment or prevention of bronchoconstriction and for reducing the exacerbations risk in asthma patients aged 18 years and above. It is claimed to be the first and only rescue medication approved for this indication in the country.

FDA Approval 130

FDA Approval Trials Development Drugs 130

XTalks

JANUARY 21, 2025

The FDA has approved Datroway (datopotamab deruxtecan-dlnk), a TROP2-directed antibody-drug conjugate (ADC) for adults with unresectable or metastatic HR-positive, HER2-negative breast cancer, developed through a global collaboration between Daiichi Sankyo and AstraZeneca. to 4,437 yen, reflecting investor confidence.

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Antibody FDA Approval HR Trials 105

Pharmaceutical Technology

MAY 22, 2023

Continued approval for the indication is dependent on verification and description of the clinical benefit of Epkinly in confirmatory trials. AbbVie research and development senior vice-president and chief scientific officer Thomas Hudson stated: “DLBCL is an aggressive cancer type that can rapidly progress and resist treatment.

FDA Approval 130

FDA Approval Clinical Trials Clinical Trials Antibody 130

XTalks

APRIL 9, 2025

The FDAs accelerated approval of Vanrafia was based on an interim analysis of the global Phase III ALIGN trial, in which atrasentan added to optimized reninangiotensin system inhibitor therapy achieved a 36.1% reduction in urine proteintocreatinine ratio versus placebo (p<0.0001).

FDA Approval 59

FDA Approval Clinical Trials Clinical Trials Trials 59

Pharmaceutical Technology

AUGUST 25, 2022

The latest approval is mainly based on positive findings from the single-arm, open-label, multicentre Phase I/II iMAGINE clinical trial of Imbruvica to treat moderate or severe cGVHD in paediatric and young adults aged one year to under 22 years. It is co-developed and co-marketed by AbbVie company, Pharmacyclics, and Janssen Biotech.

FDA Approval 130

FDA Approval Clinical Trials Clinical Trials Protein 130

Camargo

NOVEMBER 11, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Still, navigating such patent issues during drug development can be difficult, and Camargo can assist in finding a safe course. Ken Phelps.

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Development Bioequivalency Generic Drugs Production 242

XTalks

FEBRUARY 19, 2025

Deciphera leverages its proprietary switch-control kinase inhibitor platform targeting kinase activation to develop orally administered drug candidates, reflecting its commitment to innovative treatments for conditions with limited therapeutic options. TGCT is a rare, non-cancerous tumor that develops inside or near joints.

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FDA Approval Drugs Trials Clinical Trials 59

Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

XTalks

APRIL 15, 2025

Soleno Therapeutics has announced the FDA approval of Vykat XR (diazoxide choline extended-release tablets) for the treatment of hyperphagia an intense, persistent hunger in patients with Prader-Willi syndrome (PWS). The approval marks the first FDA-approved therapy specifically targeting hyperphagia in this rare genetic disorder.

FDA Approval 59

FDA Approval Clinical Trials Clinical Trials Trials 59

XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

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Gene Therapy Protein Genetic Disease Gene 105

Pharmaceutical Technology

JULY 19, 2022

The US Food and Drug Administration (FDA) has granted approval for Incyte’s Opzelura (ruxolitinib) cream 1.5% With the latest development, Opzelura has became the first treatment for repigmentation in patients with vitiligo to receive FDA approval. The findings were consistent across both trials.

FDA Approval 290

FDA Approval Clinical Trials Clinical Trials Trials 290

Pharmaceutical Technology

NOVEMBER 14, 2022

Elahere obtained the approval based on the findings on objective response rate (ORR) and duration of response (DOR) reported in the SORAYA clinical trial. Elahere is said to be the first ADC to receive FDA approval for platinum-resistant disease. The ADC comprises an FR?-binding

Antibody 246

Antibody FDA Approval Clinical Trials Clinical Trials 246