Development, Drugs and FDA Approval - Clinical Research Informer

FDA approves first-of-its-kind lupus drug

Bio Pharma Dive

JANUARY 25, 2021

Lupkynis, the first oral drug approved for lupus nephritis, comes with a high list price. Its developer, Aurinia Pharmaceuticals, expects average net revenue of roughly $65,000 per patient per year.

FDA Approval

FDA Approval Drugs Development

US FDA approves Bausch + Lomb and Novaliq’s DED treatment Miebo

Pharmaceutical Technology

MAY 19, 2023

The US Food and Drug Administration (FDA) has granted approval to Bausch + Lomb and Novaliq’s Miebo (perfluorohexyloctane ophthalmic solution) to treat the signs and symptoms of dry eye disease (DED). It is the first and only prescription eye drop to receive FDA approval to treat DED that targets tear evaporation directly.

FDA Approval

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US FDA approves Shorla’s oncology drug for T-cell leukaemia

Pharmaceutical Technology

MARCH 8, 2023

The US Food and Drug Administration (FDA) has granted approval for Shorla Oncology’s Nelarabine Injection, an oncology drug, to treat T-cell leukaemia, an aggressive blood and bone marrow cancer that progresses quickly. The company develops innovative oncology drugs with a focus on paediatric and orphan cancers.

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GPCR-targeting drugs: A renewed focus on a ubiquitous group of proteins

Bio Pharma Dive

MARCH 7, 2023

As many as a third of FDA-approved drugs target GPCRs in some fashion, with uses ranging from treating cancer to pain. But biotech startups say there is still room to develop more.

Protein

Protein FDA Approval Drugs Development

FDA approves Sanofi’s long-lasting hemophilia drug

Bio Pharma Dive

FEBRUARY 23, 2023

Sanofi and its development partner, Sobi, claim the drug’s once-a-week dosing, and its effect on blood-clotting protein levels, will help it compete in the crowded market for hemophilia A treatments.

FDA Approval

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US FDA approves Reata Pharmaceuticals’ SKYCLARYS for Friedreich’s ataxia

Pharmaceutical Technology

MARCH 2, 2023

The US Food and Drug Administration (FDA) has approved Reata Pharmaceuticals ’ oral, once-daily medication SKYCLARYS (omaveloxolone) to treat Friedreich’s ataxia patients. SKYCLARYS is the first approved treatment in Friedrich’s Ataxia and marks a milestone for drug development in this complex disease.

FDA Approval

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Symvess: Humacyte’s FDA-Approved Bioengineered Solution for Vascular Trauma

XTalks

JANUARY 7, 2025

In a major leap forward for trauma care, Humacyte has received full approval from the US Food and Drug Administration (FDA) for Symvess, a first-of-its-kind bioengineered human tissue designed to repair extremity arterial injuries. Symvess does carry risks, including thrombosis, graft rupture and infection.

FDA Approval

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5 FDA approval decisions to watch in the 4th quarter

Bio Pharma Dive

SEPTEMBER 29, 2020

The world's attention will be on the FDA as it considers initial data from coronavirus vaccine developers. But several other important drugs, including a CAR-T therapy and an Ebola antibody, will also be on the agency's agenda.

FDA Approval

FDA Approval Antibody Vaccine Vaccination

US FDA approves Ferring’s Rebyota for Clostridioides difficile infection

Pharmaceutical Technology

DECEMBER 1, 2022

The US Food and Drug Administration (FDA) has granted approval for Ferring Pharmaceuticals’ faecal microbiota product, Rebyota, to prevent Clostridioides difficile infection (CDI) recurrence in people aged 18 years and above. A live biotherapeutic, Rebyota is intended for usage following the completion of antibiotic treatment.

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Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency

XTalks

NOVEMBER 14, 2024

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. Some patients also experience distressing episodes called oculogyric crises, in which the eyes lock upward in painful spasms.

Gene Therapy

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US FDA approves Daiichi Sankyo-AstraZeneca’s Enhertu for lung cancer

Pharmaceutical Technology

AUGUST 12, 2022

The US Food and Drug Administration (FDA) has granted approval for Daiichi Sankyo and AstraZeneca ’s Enhertu (fam-trastuzumab deruxtecan-nxki) to treat unresectable or metastatic non-small cell lung cancer (NSCLC) in adults. In the trial, 1.9%

FDA Approval

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Novartis gets FDA approval of closely watched rare disease drug

Bio Pharma Dive

DECEMBER 6, 2023

The pharma is developing Fabhalta, now cleared for paroxysmal nocturnal hemoglobinuria, for several other rare, complement-driven diseases.

FDA Approval

FDA Approval Drugs Development

FDA approves Regeneron antibody drug as first Ebola virus treatment

Bio Pharma Dive

OCTOBER 14, 2020

Research for the drug, now cleared as Inmazeb, helped Regeneron jumpstart its fast development of an antibody therapy for COVID-19.

Antibody

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Open Source “Wikipedia” for Drug Discovery

Pharma Mirror

OCTOBER 4, 2021

Open source drug discovery was proposed in the past in connection with third-world diseases like tuberculosis and malaria, but it is in the context of first-world indications where it is needed most. The post Open Source “Wikipedia” for Drug Discovery appeared first on Pharma Mirror Magazine. By Kambiz Shekdar, Ph.D.

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FDA approves new antibiotic for hospital-acquired pneumonia

Bio Pharma Dive

MAY 24, 2023

The drug, called Xacduro, will be made available later this year by Innoviva Specialty Therapeutics, a new company formed from antibiotic developers Entasis and La Jolla.

FDA Approval

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US FDA approves GSK’s Jesduvroq to treat anaemia of CKD

Pharmaceutical Technology

FEBRUARY 2, 2023

The US Food and Drug Administration (FDA) has approved GlaxoSmithKline ’s (GSK) Jesduvroq (daprodustat) to treat anaemia caused by chronic kidney disease (CKD) in adults who have been on dialysis for at least four months. Jesduvroq is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI).

FDA Approval

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US FDA approves Regeneron-Sanofi’s Kevzara to treat polymyalgia rheumatica

Pharmaceutical Technology

MARCH 1, 2023

The US Food and Drug Administration (FDA) has granted approval for Regeneron Pharmaceuticals and Sanofi ’s Kevzara (sarilumab) to treat polymyalgia rheumatica (PMR) in adult patients. Developed using VelocImmune technology of Regeneron, Kevzara binds to the IL-6 receptor specifically and inhibits IL-6-mediated signalling.

FDA Approval

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Novaliq’s dry eye disease therapy receives US FDA approval

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.

FDA Approval

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US FDA approves Pfizer’s migraine nasal spray Zavzpret

Pharmaceutical Technology

MARCH 10, 2023

The US Food and Drug Administration (FDA) has granted approval for Pfizer ’s Zavzpret (zavegepant) for the acute treatment of migraine in adult patients with or without aura. Zavzpret is claimed to be the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved to treat migraine.

FDA Approval

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Seres and Nestlé Health receive FDA approval for Vowst microbiome therapy

Pharmaceutical Technology

APRIL 27, 2023

Seres Therapeutics and Nestlé Health Science have received approval from the US Food and Drug Administration (FDA) for Vowst (faecal microbiota spores, live-brpk) for preventing the recurrence of C difficile infection (CDI) in adults. It is not indicated to treat CDI.

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AstraZeneca-Daiichi Sankyo’s Enhertu gets US FDA approval for breast cancer

Pharmaceutical Technology

AUGUST 8, 2022

AstraZeneca and Daiichi Sankyo ’s Enhertu (trastuzumab deruxtecan) has received expanded approval from the US Food and Drug Administration (FDA) to treat adults with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer.

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US FDA approves Eisai-Biogen’s antibody for Alzheimer’s

Pharmaceutical Technology

JANUARY 9, 2023

Eisai and Biogen have received approval for their antibody Leqembi (lecanemab-irmb) , 100mg/mL injection for intravenous use, from the US Food and Drug Administration (FDA) under the Accelerated Approval Pathway to treat Alzheimer’s disease (AD).

FDA Approval

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FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

Pharmaceutical Technology

SEPTEMBER 19, 2022

The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio ’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. The treatment is indicated for usage in boys aged four to 17 years with CALD.

FDA Approval

FDA Approval In-Vivo Gene Therapy Clinical Trials

FDA approves new RSV drug to protect infants, toddlers

Bio Pharma Dive

JULY 17, 2023

Developers AstraZeneca and Sanofi expect to make the preventive antibody treatment, called Beyfortus, available ahead of the upcoming cold season.

FDA Approval

FDA Approval Antibody Drugs Development

Journey Medical’s Emrosi Wins FDA Approval for Rosacea, Could Rival Galderma’s Oracea

XTalks

NOVEMBER 7, 2024

Galderma’s doxycycline drug Oracea (doxycycline 40 mg capsules) is the current standard-of-care treatment for rosacea. Emrosi’s approval was based on data from a pair of Phase III clinical trials for the treatment of rosacea. Other studies suggest that Helicobacter pylori bacteria infections may also cause rosacea.

FDA Approval

FDA Approval Dermatology Bacteria Clinical Trials

BMS receives US FDA approval for CAR T cell therapy to treat B-cell lymphoma

Pharmaceutical Technology

JUNE 27, 2022

Bristol Myers Squibb (BMS) has received approval from the US Food and Drug Administration (FDA) for its CD19-directed chimeric antigen receptor (CAR) T cell therapy Breyanzi (lisocabtagene maraleucel) to treat relapsed or refractory large B-cell lymphoma (LBCL) in adult patients.

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Vertex gains FDA approval for expanded use of CF drug Trikafta

Pharmaceutical Technology

APRIL 27, 2023

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) to expand the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to treat cystic fibrosis (CF) in children aged between two and five years with certain mutations.

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Sanofi’s Qfitlia Gets FDA Approval as First siRNA Therapy for Hemophilia

XTalks

APRIL 1, 2025

Some individuals with hemophilia A may develop inhibitors against factor VIII, while those with hemophilia B can develop inhibitors against factor IX. Clinical Trials Backing Qfitlias Approval Two Phase III studies involving adult and pediatric male patients with hemophilia A or B supported Qfitlias FDA approval.

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FDA extends review of GSK’s myelofibrosis drug

Pharmaceutical Technology

JUNE 19, 2023

UK-based pharmaceutical giant GSK has announced that the US Food and Drug Administration (FDA) has extended the review period of its new drug application (NDA) for the rare bone cancer drug momelotinib by three months. The drug is not currently approved in any markets globally.

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Krystal Biotech receives FDA approval for DEB topical gene therapy

Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. VYJUVEK is designed to address the underlying genetic cause of the disease.

Gene Therapy

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Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment

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

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AGEPHA Pharma’s LODOCO gets US FDA approval for cardiovascular disease

Pharmaceutical Technology

JUNE 21, 2023

AGEPHA Pharma has received approval from the US Food and Drug Administration (FDA) for LODOCO (colchicine, 0.5 mg tablet) to treat cardiovascular disease.

FDA Approval

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FDA Approves New Use for Ozempic to Reduce the Risks of Kidney Disease

XTalks

JANUARY 29, 2025

The FDA has approved Novo Nordisks Ozempic (semaglutide) for reducing the risk of kidney failure, progression of kidney disease and cardiovascular death in adults with type 2 diabetes and chronic kidney disease (CKD). The approval marks Ozempic as the first GLP-1 receptor agonist (GLP-1 RA) authorized in a kidney disease indication.

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Transforming drug development with AI

pharmaphorum

FEBRUARY 14, 2024

Discover how artificial intelligence (AI) is revolutionising the drug development process and transforming the way FDA-approved drugs are produced, ensuring compliance and optimising the supply chain. Learn more about the future of AI in pharmaceuticals.

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US FDA approves Luye Pharma’s schizophrenia treatment Rykindo

Pharmaceutical Technology

JANUARY 16, 2023

The US Food and Drug Administration (FDA) has approved Luye Pharma Group’s Rykindo (risperidone) as an extended-release injectable suspension to treat schizophrenia in adult patients. Rykindo has also been approved as a single agent or as adjunctive therapy to lithium or valproate to treat bipolar I disorder in these patients.

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Top 30 New Medical Devices of 2024

XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Agent drug-coated balloon. The balloons outer surface is coated with the drug paclitaxel, a safe and effective measure to prevent the arteries from narrowing again.

FDA Approval

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Gilead wins patent battle with US government over HIV PrEP drugs

Pharmaceutical Technology

MAY 10, 2023

Gilead Sciences has emerged victorious in a legal battle with the US government over patents surrounding the HIV pre-exposure prophylaxis (PrEP) drugs Descovy and Truvada following a federal jury’s verdict on May 9. This marks a significant development in a tumultuous dispute between the pharmaceutical company and the US government.

Drugs

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US FDA grants Orphan Drug status to Avacta’s drug for soft tissue sarcoma

Pharmaceutical Technology

SEPTEMBER 6, 2022

The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to Avacta Group’s lead pre|CISION drug candidate, AVA6000, to treat soft tissue sarcoma. This status offers the drug developer some incentives, such as market exclusivity for seven years on obtaining FDA approval.

Drugs

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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans. So far this year, 176 drugs have already received this designation.

Drugs

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Romvimza (Vimseltinib) for TGCT: FDA Approves Oral CSF1-Targeted Drug

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.

FDA Approval

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FDA approves first biosimilar to top-selling eye drug Lucentis

Bio Pharma Dive

SEPTEMBER 20, 2021

The approval of Byooviz, developed by Samsung Bioepis and set to be sold by Biogen, comes amid a slowdown in biosimilar clearances from the agency.

FDA Approval

FDA Approval Drugs Development

Lilly’s Jaypirca received FDA approval to treat MCL

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). Furthermore, the company intends to commercialise the drug in the country within a few weeks.

FDA Approval

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US FDA launches lupus consortium to confront drug development challenges

Pharmaceutical Technology

MARCH 30, 2023

The US Food and Drug Administration (FDA)’s Center for Drug Evaluation and Research and the Lupus Accelerating Breakthroughs Consortium (Lupus ABC) have launched the Lupus Research Alliance (LRA) in a bid to advance research for the condition. Asian, Hispanic, and Native American women are also at higher risk.

Development

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Top 10 Heart and Vascular Medications Based on Recent Sales Data

XTalks

JANUARY 22, 2025

From blockbuster anticoagulants like Eliquis (apixaban) and Xarelto (rivaroxaban) to life-saving treatments such as Entresto (sacubitril/valsartan) and Brilinta (ticagrelor), each of these drugs has made a significant impact in the cardiovascular space. Partnered with Pfizer, the drug continues to show strong market performance.

Sales

Sales FDA Approval Drugs Marketing

FDA approves first-of-its-kind lupus drug

US FDA approves Bausch + Lomb and Novaliq’s DED treatment Miebo

Webinars

Trending Sources

US FDA approves Shorla’s oncology drug for T-cell leukaemia

Webinars

GPCR-targeting drugs: A renewed focus on a ubiquitous group of proteins

FDA approves Sanofi’s long-lasting hemophilia drug

US FDA approves Reata Pharmaceuticals’ SKYCLARYS for Friedreich’s ataxia

Symvess: Humacyte’s FDA-Approved Bioengineered Solution for Vascular Trauma

5 FDA approval decisions to watch in the 4th quarter

US FDA approves Ferring’s Rebyota for Clostridioides difficile infection

Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency

US FDA approves Daiichi Sankyo-AstraZeneca’s Enhertu for lung cancer

Novartis gets FDA approval of closely watched rare disease drug

FDA approves Regeneron antibody drug as first Ebola virus treatment

Open Source “Wikipedia” for Drug Discovery

FDA approves new antibiotic for hospital-acquired pneumonia

US FDA approves GSK’s Jesduvroq to treat anaemia of CKD

US FDA approves Regeneron-Sanofi’s Kevzara to treat polymyalgia rheumatica

Novaliq’s dry eye disease therapy receives US FDA approval

US FDA approves Pfizer’s migraine nasal spray Zavzpret

Seres and Nestlé Health receive FDA approval for Vowst microbiome therapy

AstraZeneca-Daiichi Sankyo’s Enhertu gets US FDA approval for breast cancer

US FDA approves Eisai-Biogen’s antibody for Alzheimer’s

FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

FDA approves new RSV drug to protect infants, toddlers

Journey Medical’s Emrosi Wins FDA Approval for Rosacea, Could Rival Galderma’s Oracea

BMS receives US FDA approval for CAR T cell therapy to treat B-cell lymphoma

Vertex gains FDA approval for expanded use of CF drug Trikafta

Sanofi’s Qfitlia Gets FDA Approval as First siRNA Therapy for Hemophilia

FDA extends review of GSK’s myelofibrosis drug

Krystal Biotech receives FDA approval for DEB topical gene therapy

Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment

AGEPHA Pharma’s LODOCO gets US FDA approval for cardiovascular disease

FDA Approves New Use for Ozempic to Reduce the Risks of Kidney Disease

Transforming drug development with AI

US FDA approves Luye Pharma’s schizophrenia treatment Rykindo

Top 30 New Medical Devices of 2024

Gilead wins patent battle with US government over HIV PrEP drugs

US FDA grants Orphan Drug status to Avacta’s drug for soft tissue sarcoma

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Romvimza (Vimseltinib) for TGCT: FDA Approves Oral CSF1-Targeted Drug

FDA approves first biosimilar to top-selling eye drug Lucentis

Lilly’s Jaypirca received FDA approval to treat MCL

US FDA launches lupus consortium to confront drug development challenges

Top 10 Heart and Vascular Medications Based on Recent Sales Data

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