XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. Duvyzat received Priority Review, Orphan Drug and Rare Pediatric Disease designations from the FDA.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

XTalks

NOVEMBER 26, 2024

BridgeBio’s leadership, including Neil Kumar, emphasizes the significance of Attruby’s approval as a cornerstone in their mission to develop therapies for genetic diseases. Looking ahead, the company plans to pursue regulatory approvals in Japan and Brazil, further expanding access to this life-changing treatment.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

Pharmaceutical Technology

AUGUST 18, 2022

bluebird bio CEO Andrew Obenshain said: “The FDA approval of Zynteglo offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation and unlocks new possibilities in their daily lives.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

Pharmaceutical Technology

SEPTEMBER 23, 2022

While a handful of therapies have launched since then, late-stage pipeline therapies that are currently in development reveal that drug developers are exploring a broad set of mechanisms of action (MOAs), many of which are innovative, to tackle DES.

Development 147

Development Drugs Clinical Trials Clinical Trials 147

Fierce Pharma

DECEMBER 13, 2024

Even though AI-designed drugs arent yet a household term for FDA-approved, commercially available therapies, they are a reality in clinical development pipelines.

FDA Approval 82

FDA Approval Clinical Development Drugs Development 82

BioSpace

NOVEMBER 2, 2021

Food and Drug Administration accepted Investigational New Drug Applications for multiple companies, clearing the way for clinical development. This week, the U.S.

FDA Approval 77

FDA Approval Drugs Clinical Development Development 77

Pharmaceutical Technology

FEBRUARY 8, 2023

In 2020, the FDA released a guidance document for the industry with recommendations for sponsors developing gene therapies for rare diseases. This document delved into aspects related to manufacturing, preclinical, and clinical trial design for all phases of clinical development.

Gene Therapy 264

Gene Therapy Gene Trials Clinical Trials 264

BioPharma Reporter

AUGUST 13, 2024

The FDA has given the green light to Lymphir, the first immunotherapy for the treatment of cutaneous T-cell lymphoma (CTCL) to get approval in over five years.

FDA Approval 116

FDA Approval Clinical Development Development 116

Outsourcing Pharma

DECEMBER 18, 2023

The oral treatment belzutifan, developed by MSD (known as Merck in the US) and branded as Welireg, has received the green light from the US Food and Drug Administration (FDA) for the treatment of an advanced form of renal cell carcinoma (RCC).

FDA Approval 92

FDA Approval Branding Development Drugs 92

Outsourcing Pharma

JULY 19, 2024

Phathom Pharmaceuticals, Inc.dedicated to developing and commercializing treatments for gastrointestinal (GI) diseases, announced today that the US Food and Drug Administration (FDA) has approved Voquenza (vonoprazan) 10mg tablets for the relief of heartburn associated with non-erosive gastroesophageal reflux disease (non-erosive GERD) in adults.

FDA Approval 88

FDA Approval Development Drugs Clinical Development 88

Outsourcing Pharma

DECEMBER 14, 2023

Mission Therapeutics, a clinical-stage biotech developing first-in-class therapeutics targeting mitophagy, has received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) to proceed with a phase 2 trial for its kidney treatment MTX652.

FDA Approval 96

FDA Approval Drugs Trials Development 96

XTalks

DECEMBER 10, 2021

Zavegepant has an advantage over the two as it’s the only CGRP receptor antagonist in both intranasal spray and oral pill formulations that is in clinical development for the treatment of acute or episodic migraines.

FDA Approval 98

FDA Approval Trials Drugs Sales 98

BioPharma Reporter

AUGUST 2, 2021

The drug, intended for adult patients with moderate-to-severe systemic lupus erythematosus, is the only new therapy approved for SLE in more than a decade.

FDA Approval 98

FDA Approval Drugs Clinical Development Development 98

Outsourcing Pharma

DECEMBER 19, 2023

The US Food and Drug Administration (FDA) has approved a topical foam developed by Arcutis Biotherapeutics and branded as Zoryve for the skin condition seborrheic dermatitis in people aged nine years and up.

FDA Approval 81

FDA Approval Branding Development Drugs 81

Outsourcing Pharma

JANUARY 25, 2022

The agency has given the go-ahead on an IND application for a study evaluating Ananda Scientificâs Nantheia ATL5, a candidate for treating opioid use disorder.

FDA Approval 102

FDA Approval Clinical Trials Clinical Trials Trials 102

pharmaphorum

AUGUST 10, 2020

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). The FDA approved Evrysdi for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older.

FDA Approval 59

FDA Approval Drugs Gene Therapy Protein 59

Outsourcing Pharma

NOVEMBER 13, 2023

A small molecule, oral targeted therapy for adults with metastatic colorectal cancer (CRC) has been approved by the US Food and Drug Administration (FDA).

FDA Approval 88

FDA Approval Drugs Clinical Development Development 88

Delveinsight

SEPTEMBER 9, 2021

A collaboration valued more than USD 3 billion, Genentech, a member of the Roche Group, declared it will collaborate with Adaptimmune for developing and commercializing allogeneic T-cell therapies to treat multiple cancer indications. TransMedics receives FDA approval for its OCS heart system.

FDA Approval 73

FDA Approval Development Research Production 73

Pharmaceutical Technology

JUNE 7, 2023

In 2017, the therapy received FDA approval for prophylaxis of CMV infection and disease in adult CMV-seropositive recipients of an allogeneic hematopoietic stem cell transplant. The drug is given once a day as an oral tablet or as an injection for intravenous infusion.

FDA Approval 130

FDA Approval Clinical Development Drugs Medicine 130

Outsourcing Pharma

SEPTEMBER 30, 2020

The companyâs Phase II trial of ultramicronized PEA has received FDA approval and expects to begin dosing patientparticipants as early as next month.

Trials 119

Trials FDA Approval Clinical Development Development 119

pharmaphorum

OCTOBER 5, 2020

The FDA has approved Opdivo (nivolumab) and Yervoy (ipilimumab), as the first and only immunotherapy for previously untreated unresectable malignant pleural mesothelioma. The post FDA approves BMS’ Opdivo and Yervoy combination in first-line mesothelioma appeared first on.

FDA Approval 59

FDA Approval Trials Clinical Development Development 59

Outsourcing Pharma

SEPTEMBER 18, 2023

The small molecule drug Ojjaara (momelotinib), developed by GlaxoSmithKline, has become the first U.S. Food and Drug Administration (FDA)-approved treatment for patients with the blood cancer myelofibrosis and anemia.

FDA Approval 91

FDA Approval Development Drugs Clinical Development 91

Pharmaceutical Technology

MARCH 1, 2023

The Covid-19 pandemic led to massive developments and scientific advances within the field of messenger ribonucleic acid (mRNA) vaccines. Pfizer /BioNTech and Moderna’s mRNA-based vaccines were the first to receive emergency use authorisation (EUA) out of all other Covid-19 vaccines under development in late 2020.

RNA 147

RNA Vaccine Vaccination Genetics 147

The Pharma Data

NOVEMBER 30, 2020

Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. is undertaking clinical trials for NVX-CoV2373, its vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. GAITHERSBURG, Md., Source link.

Clinical Development 52

Clinical Development Vaccine Vaccination Development 52

Outsourcing Pharma

DECEMBER 14, 2023

Mission Therapeutics, a clinical-stage biotech developing first-in-class therapeutics targeting mitophagy, has received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) to proceed with a phase 2 trial for its kidney treatment MTX652.

FDA Approval 78

FDA Approval Drugs Trials Development 78

pharmaphorum

SEPTEMBER 3, 2020

UK biotech Compass Pathways is planning a $100 million US IPO as it seeks to further develop its depression treatment based on psilocybin – an active ingredient that can be found in magic mushrooms.

Drugs 116

Drugs Life Science Clinical Development Regulation 116

pharmaphorum

SEPTEMBER 21, 2020

COMP360 is a crystalline formulation of psilocybin in phase 2b clinical development in patients who have not responded to standard therapy for depression. UK-based Compass has a patented synthetic version of psilocybin, the active ingredient in magic mushrooms, and raised around $127.5

Medicine 134

Medicine FDA Approval Clinical Trials Clinical Trials 134

pharmaphorum

MARCH 23, 2022

Merck & Co’s chief medical officer and head of clinical development Dr Roy Baynes has announced his retirement from the company to take up a new role alongside his former colleague Roger Perlmutter at biotech Eikon Therapeutics. billion last year.

Clinical Development 98

Clinical Development FDA Approval Vaccine Vaccination 98

Pfizer

AUGUST 3, 2022

Pfizer to Discontinue Development Program for PF-07265803 for LMNA-Related Dilated Cardiomyopathy. Pfizer to Discontinue Development Program for PF-07265803 for LMNA-Related Dilated Cardiomyopathy. Based on these results, the Phase 3 trial and further development of PF-07265803 will be discontinued. deliesschef.

Development 101

Development Vaccine Vaccination Production 101

Outsourcing Pharma

FEBRUARY 19, 2024

Patients in the US with epidermal growth factor receptor mutated (EGFRm) advanced lung cancer can now receive an important new treatment option delaying disease progression by nearly nine additional months.

FDA Approval 86

FDA Approval Clinical Development Development 86

Outsourcing Pharma

NOVEMBER 16, 2023

The first and only enzyme replacement therapy for the treatment of a very rare and complex blood disorder has been approved by the US Food and Drug Administration.

FDA Approval 85

FDA Approval Drugs Clinical Development Development 85

XTalks

NOVEMBER 5, 2024

Proceeds from the IPO will fund preclinical and clinical development for Polyrizon’s innovative hydrogel product candidates, supporting further research and development (R&D). Trading began on the Nasdaq Capital Market under the ticker “PLRZ” on October 29, 2024.

Drug Delivery 111

Drug Delivery Drugs Immune Response Allergies 111

Outsourcing Pharma

JUNE 22, 2022

The company reports it has met the enrollment target for a trial of its sonic beam therapy, one step closer to submitting for FDA approval of the product.

FDA Approval 98

FDA Approval Production Trials Clinical Development 98

pharmaphorum

MAY 30, 2022

Despite this, the first-ever FDA-approved KRAS G12C inhibitor has demonstrated that RAS is in fact druggable and that drugging this protein unlocks a world of successful therapeutic interventions. Continuing to advance valuable combination therapies into the clinic, and finally, crack resistance to RAS targeted therapeutics.

Development 52

Development Drugs FDA Approval Clinical Trials 52

Pharmaceutical Technology

OCTOBER 25, 2022

Understanding both the significant competitive advantages that FDC products can offer, and the difficulties and potential pitfalls associated with development and manufacturing of FDC drugs in oral solid dosage forms is a critical first step for companies exploring this approach. Pharmaceutical companies – and the FDA – are embracing FDCs.

Manufacturing 130

Manufacturing Production Packaging Packaging 130

pharmaphorum

JANUARY 5, 2022

Having already brought their mRNA-based CVID-19 vaccine Comirnaty to market at breakneck speed, Pfizer and BioNTech are hoping to fast-track a vaccine for shingles based on the same technology platform through clinical development, with trials due to start later this year.

Vaccine 105

Vaccine Vaccination Sales Clinical Development 105

pharmaphorum

MAY 13, 2021

There are already several drugs on the market to treat Duchenne, notably from Sarepta Therapeutics which has three FDA-approved drugs that use “exon skipping” technology. Sarepta is also developing a DMD gene therapy, although it’s a step behind Pfizer in mid-stage clinical development.

Gene Therapy 102

Gene Therapy Gene Trials Protein 102