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.

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FDA Approval Gene Therapy Genetic Disease Gene 130

XTalks

NOVEMBER 30, 2023

Pfizer spinout SpringWorks Therapeutics’ Ogsiveo (nirogacestat) has received US Food and Drug Administration (FDA) approval for the treatment of desmoid tumors, an ultra-rare subtype of non-cancerous soft tissue sarcomas that can cause severe pain and disfigurement.

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FDA Approval Trials Clinical Trials Clinical Trials 101

XTalks

FEBRUARY 6, 2023

GlaxoSmithKline LLC (GSK) recently announced that the US Food and Drug Administration (FDA) approved Jesduvroq (daprodustat), a new once-a-day oral treatment for anemia due to chronic kidney disease (CKD). The approval is currently only for adult patients who have been undergoing dialysis for at least four months.

FDA Approval 97

FDA Approval Clinical Trials Clinical Trials Trials 97

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. The most commonly reported adverse reactions in the clinical trial were pruritus (itching) and pain at the wound application site, occurring in 7.3 percent of cases.

FDA Approval 111

FDA Approval Gene Therapy Gene Trials 111

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.

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FDA Approval Clinical Trials Clinical Trials Trials 189

XTalks

AUGUST 7, 2023

Ycanth (cantharidin) topical solution, developed by Verrica Pharmaceuticals Inc., has received US Food and Drug Administration (FDA) approval for treating molluscum contagiosum in adult and pediatric patients aged two years and older in the US. What is Molluscum Contagiosum?

FDA Approval 98

FDA Approval Dermatology Clinical Trials Clinical Trials 98

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. In the US, more than 30 million people live with a rare disease.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

Delveinsight

AUGUST 13, 2021

Clinical trials are an essential part of the drug development process. Worldwide, more than 2 million clinical trials are registered and the number is steadily increasing over the past several years. The conduct of trials was not possible due to the risk of catching coronavirus. Why the Need?

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Clinical Trials Clinical Trials Trials Drugs 98

XTalks

APRIL 25, 2024

Now we recognize that over the last ten years or so there have been major developments in pharmacotherapy for the management of different etiologies of heart failure,” says Dr. Nicholas Alp, MD, PhD, FACC, FRCP, Vice President of the Medical Department at Medpace.

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Development Trials Clinical Trials Clinical Trials 111

XTalks

DECEMBER 14, 2023

Evive Biotech, a leading biopharmaceutical company based in China, and Acrotech Biopharma, a subsidiary of Aurobindo Pharma USA in New Jersey, have jointly announced a major achievement with the US Food and Drug Administration’s (FDA) approval of Ryzneuta (efbemalenograstim alfa) for treating chemotherapy-induced neutropenia (CIN).

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Trials 52

XTalks

JULY 25, 2023

“The approval of Cyfendus vaccine is symbolic of Emergent’s longstanding partnership with the US government and our shared commitment to helping protect public health,” said Dr. Kelly Warfield, Emergent’s senior vice president, science and development, in the company’s press release. What Is Anthrax? How Does Cyfendus Work?

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FDA Approval Vaccination Vaccine Antibody 98

XTalks

DECEMBER 22, 2022

Neilan has contributed to the rare disease community through his comprehensive work in the rare disease space that has included research, academia, medical practice and clinical trial design and execution. Can you tell us a bit about the work you are doing at NORD and how it impacts clinical trials for rare diseases?

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Clinical Trials Clinical Trials Trials Clinical Research 98

XTalks

OCTOBER 4, 2022

Rolvedon is indicated to lower the chance of infection from febrile neutropenia (the development of fever, often with other signs of infection, in patients with a very low number of neutrophil granulocytes in the blood) in adult patients with non-myeloid malignancies on myelosuppressive anti-cancer drugs. How Does Rolvedon Work?

FDA Approval 98

FDA Approval Clinical Trials Clinical Trials Trials 98

XTalks

MARCH 9, 2023

The disease develops between the ages of 20 to 30 and progresses over time, often leading to end-stage kidney failure due to glomerular disease. Results for this endpoint are expected in the final quarter of 2023 and are expected to support the traditional approval of Filspari.

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

Pharmaceutical Technology

AUGUST 16, 2022

Q32 Bio has signed a partnership and option agreement with Horizon Therapeutics for developing ADX-914 to treat autoimmune ailments. According to the deal, Horizon will provide the funding to develop the therapy until the conclusion of its two Phase II clinical trials. A fully human anti-IL-7R?

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Development Clinical Trials Clinical Trials FDA Approval 130

pharmaphorum

JULY 8, 2022

The COVID-19 pandemic has catalysed significant changes in the way pharma develops drugs, particularly in the clinical trial space. Hybrid or decentralised clinical trials (DCTs) have gained traction as technology, infrastructure and knowledge have evolved to support their use. Source: Izmailova et al, 2017.

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Clinical Trials Clinical Trials Trials Regulation 79

XTalks

OCTOBER 4, 2022

XTALKS WEBINAR: Time for Change in NASH: How Non-Invasive Biomarkers are Driving a Paradigm Shift in Clinical Trials and Clinical Care. Outcomes of Clinical Trials of Terlipressin. The clinical trial results were published in the New England Journal of Medicine in March 2021. percent (vs. percent (vs.

FDA Approval 98

FDA Approval Clinical Trials Clinical Trials Trials 98

XTalks

JANUARY 4, 2023

MediWound, a biopharmaceutical company focused on biotherapeutic solutions for tissue repair and regeneration, announced that the US Food and Drug Administration (FDA) approved their orphan biological product NexoBrid (anacaulase-bcdb) for the removal of eschar in adults with deep partial-thickness and/or full-thickness thermal burns.

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FDA Approval Clinical Trials Clinical Trials Contamination 98

XTalks

MAY 5, 2022

There are four FDA-approved ADHD-specific nonstimulant drugs, namely Eli Lilly’s Strattera (atomoxetine), Shionogi’s Kapvay (clonidine), Takeda’s Intuniv (guanfacine) and now Supernus’ Qelbree (viloxazine). In 2002, Strattera became the first FDA-approved nonstimulant drug to treat ADHD in adults.

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

JUNE 26, 2023

Obtaining FDA approval for our SurVeil DCB is one of the most important achievements in Surmodics’ history,” said Gary Maharaj, president and CEO of Surmodics, in the company’s press release. “It SurVeil has an edge over IN.PACT as it is coated with a lower dose of the drug.

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FDA Approval Drugs Sales In-Vitro 96

Pharmaceutical Technology

MARCH 17, 2023

The liquid formulations of Tafinlar and Mekinist also received approval for ease of administration across several approved indications. This marks the first time a BRAF/MEK inhibitor has been developed in a formulation that is suitable for these patients as young as one year.

FDA Approval 130

FDA Approval Clinical Trials Clinical Trials Trials 130

XTalks

MAY 2, 2022

For Mycovia Pharmaceuticals , Vivjoa is the company’s first FDA-approved drug in its pipeline of novel treatments for fungal infections. North Carolina-based Mycovia is an emerging biopharmaceutical company devoted to recognizing and empowering people living with unmet medical needs through the development of novel therapies.

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

MARCH 28, 2024

The US Food and Drug Administration (FDA) has given the nod to Winrevair (sotatercept-csrk injection, 45mg, 60mg) for the treatment of adults with pulmonary arterial hypertension (PAH) to increase exercise capacity, improve WHO (World Health Organization) functional class (FC) and reduce the risk of clinical worsening events.

FDA Approval 81

FDA Approval Clinical Trials Clinical Trials Trials 81

Clinical Trial Podcast

DECEMBER 29, 2022

In this episode, I’m excited for you to learn about Heart Failure Clinical Trials for medical devices. Sonna is currently the President at NuPulse Inc, an early stage medical device company responsible for developing the first minimally invasive, long-term, ambulatory counterpulsation device that works in sync with the heart.

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Clinical Trials Clinical Trials Trials Clinical Supply 52

pharmaphorum

OCTOBER 25, 2021

Hard on the heels of some impressive clinical trial results, Luminopia has won FDA approval for its digital therapeutic (DTx) for amblyopia or lazy eye in children that is based on watching TV shows. The post Luminopia scores FDA approval for TV-based amblyopia therapy appeared first on.

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FDA Approval Clinical Trials Clinical Trials Trials 104

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 95

FDA Approval Clinical Trials Clinical Trials Trials 95

XTalks

NOVEMBER 10, 2023

The disorder usually develops in infancy or early childhood but can also appear in adulthood, including during pregnancy, in some cases. Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.”

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FDA Approval Gene Therapy Gene Clinical Trials 59

Drug Discovery World

JUNE 6, 2023

Today’s approval is a monumental step forward in delivering on Pfizer’s commitment to help alleviate the significant burden of RSV in higher-risk populations, which includes older adults,” said Annaliesa Anderson, Senior Vice President and Chief Scientific Officer, Vaccine Research and Development, Pfizer.

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FDA Approval Vaccination Vaccine Clinical Trials 52

Drug Discovery World

OCTOBER 11, 2023

Collaborators Boehringer Ingelheim and CDR-Life have commenced a Phase I evaluation of BI 771716, their antibody fragment-based treatment developed to preserve the vision of people with geographic atrophy (GA).

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Clinical Trials Clinical Trials Antibody Trials 52

Drug Discovery World

MAY 15, 2023

The US Food and Drug Administration (FDA) has given the go-ahead to CytoAgents to initiate a US Phase Ib/IIa clinical trial investigating CTO1681 for cytokine release syndrome (CRS) in lymphoma patients receiving CAR-T cell therapy.

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FDA Approval Clinical Trials Clinical Trials Trials 52

pharmaphorum

FEBRUARY 1, 2022

Across the industry, pharma companies are turning to AI and real-world data to address many of the challenges of running clinical trials. Can the combined potential of new AI technologies and real-world patient data hold the key to overcoming the challenges in clinical trial design that have historically led to trial failure?

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Clinical Trials Clinical Trials Trials Engineer 92

Drug Discovery World

AUGUST 23, 2023

Pfizer’s Abrysvo (Respiratory Syncytial Virus Vaccine) was approved for use at 32-36 weeks gestational age of pregnancy to prevent lower respiratory tract disease (LRTD) and severe LRTD caused by RSV in infants from birth to six months of age.

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FDA Approval Vaccination Vaccine Clinical Trials 52

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. Novaliq’s CyclASol (0.1%

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Development Drugs Clinical Trials Clinical Trials 147

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Regulatory Approvals for CTC-Based Assays in Oncology.

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Clinical Trials Clinical Trials Trials FDA Approval 98

XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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FDA Approval Manufacturing Trials Clinical Trials 52

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

JANUARY 6, 2023

Last week, the FDA approved a new infusible medication from TG Therapeutics for adults with relapsing forms of multiple sclerosis (MS), which include clinically isolated syndrome (CIS), relapsing-remitting (RR) MS, and active secondary progressive MS (SPMS). Briumvi (ublituximab-xiiy) Clinical Trial Results.

FDA Approval 94

FDA Approval Clinical Trials Clinical Trials Antibody 94