XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease,” said Michael J.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

Pharmaceutical Technology

JUNE 15, 2023

This approval has been granted for ALE.C04 both as a monotherapy and in combination with pembrolizumab in a first-in-human clinical study in recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). The planned Phase I/II clinical trial in HNSCC will begin in the second half of 2023.

FDA Approval 130

FDA Approval Antibody Clinical Trials Clinical Trials 130

XTalks

OCTOBER 16, 2024

Hympavzi is the first anti-tissue factor pathway inhibitor (anti-TFPI) approved in the US for hemophilia A or B, and it’s also the first hemophilia therapy to be administered via a pre-filled auto-injector pen. Hympavzi reduces the activity of TFPI, a naturally occurring anticoagulation protein.

FDA Approval 105

FDA Approval Trials Clinical Trials Clinical Trials 105

XTalks

SEPTEMBER 14, 2022

Daxxify was FDA-approved for similar cosmetic purposes as Botox and other neuromodulators like Dysport and Xeomin. Used for both cosmetic and therapeutic cases, Botox is a US Food and Drug Administration (FDA)-approved injection of botulinum toxin, a neurotoxic protein that can effectively paralyze the facial muscles.

Botox 98

Botox FDA Approval Cosmetics Clinical Trials 98

Pharmaceutical Technology

OCTOBER 25, 2022

Based on positive data from the open-label, international, randomised, multicentre Phase III HIMALAYA clinical trial, the regulatory agency granted the approval. A human monoclonal antibody, Imfinzi attaches to the PD-L1 protein and hinders the PD-L1 interaction with the PD-1 and CD80 proteins.

Protein 173

Protein Immune Response Antibody FDA Approval 173

XTalks

MARCH 9, 2023

The approval was granted for its use as a non-immunosuppressive therapy for the reduction of proteinuria (protein in urine) in IgA nephropathy in adults. Filspari is the first single molecule dual endothelin angiotensin receptor antagonist (DEARA) approved for this indication. g/g and be at risk of rapid disease progression.

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

OCTOBER 4, 2022

“Rolvedon’s approval marks Spectrum’s transformation to a commercial-stage company with the opportunity to compete in a $2 billion dollar market, and offers a unique value proposition,” said Tom Riga, President and Chief Executive Officer of Spectrum Pharmaceuticals, in the company’s press release. Clinical Trials and Commercialization.

FDA Approval 98

FDA Approval Clinical Trials Clinical Trials Trials 98

XTalks

OCTOBER 24, 2024

Amgen’s Otezla (apremilast) continues to expand its reach, with the US Food and Drug Administration (FDA) approving its use for children and adolescents with moderate to severe plaque psoriasis in 2024. This drug may significantly improve psoriasis care by offering effective long-term control with fewer side effects.

Research 105

Research Dermatology Clinical Trials Clinical Trials 105

XTalks

AUGUST 7, 2023

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. Formerly known as VP-102, Ycanth is the first cantharidin formulation approved for this purpose. Another noteworthy contender is Novan Inc.’s

FDA Approval 98

FDA Approval Dermatology Trials Clinical Trials 98

XTalks

APRIL 15, 2025

Hemophilia occurs when a clotting protein is missing or deficient, causing prolonged bleeding that can lead to joint damage and chronic pain. Despite these burdens, fewer than 5% of hemophilia clinical trials include female participants. In a cohort of 45 post-menarcheal girls in China, 46.7%

Gene Therapy 66

Gene Therapy Gene Clinical Trials Clinical Trials 66

XTalks

MAY 26, 2022

The US FDA approved Dupixent (dupilumab) developed by Regeneron as the first treatment for eosinophilic esophagitis, a chronic immune disorder that damages the esophagus. These proteins cause the type 2 inflammation characteristic of EoE and related allergic diseases like asthma and atopic dermatitis (both treated with Dupixent).

FDA Approval 105

FDA Approval Antibody Clinical Trials Clinical Trials 105

XTalks

MAY 4, 2022

Bristol Myers Squibb (BMS) was granted US Food and Drug Administration (FDA) approval for its highly awaited cardiac drug Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (HCM). The FDA approved 2.5 BMS had targeted Camzyos in its acquisition of MyoKardia in 2020 for $13.1

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

JULY 25, 2023

Cyfendus functions by stimulating the production of antibodies targeted against the protective antigen protein. Efficacy of Cyfendus The FDA approval of the Cyfendus anthrax vaccine is grounded in a series of studies supported by the US government and carried out by Emergent. Additionally, at Day 64, 86.6

FDA Approval 98

FDA Approval Vaccine Vaccination Antibody 98

XTalks

AUGUST 19, 2022

Beta thalassemia is a rare inherited blood disorder caused by mutations in the beta-globin gene leading to a reduced amount, or complete absence, of the beta chains of the oxygen-carrying hemoglobin protein in red blood cells. XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

Pharmaceutical Technology

JANUARY 22, 2023

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. However, while only around 34% of the approvals in 2018 were for orphan drugs, 54% new approvals in 2022 were for drugs to treat rare diseases. In clinical trials, the drug showed a 3.8-point

Drugs 317

Drugs Gene Hormones Clinical Trials 317

XTalks

DECEMBER 29, 2023

. “The FDA’s Priority Review designation for this application underscores the urgency to provide new treatment options for patients with advanced SCLC who have progressed following treatment with platinum-based chemotherapy,” said David M.

Clinical Trials 119

Clinical Trials Clinical Trials FDA Approval Trials 119

XTalks

JANUARY 3, 2025

For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. In clinical trials, it showed up to an 86 percent reduction in triglyceride levels.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

XTalks

MARCH 18, 2025

Incytes recent announcement on the topline results from two Phase III clinical trials of povorcitinib in patients with hidradenitis suppurativa has stirred both hope and caution in the market. By inhibiting this protein, povorcitinib reduces the inflammatory signals that contribute to abscess and nodule formation.

Trials 59

Trials Clinical Trials Clinical Trials Hormones 59

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

Pharmaceutical Technology

DECEMBER 23, 2022

This approval is backed by findings from the multicentre, double-blinded, placebo-controlled, international Phase II/III CAPELLA clinical trial of lenacapavir plus an optimised background regimen. Subcutaneous lenacapavir’s antiviral activity administered every six months was analysed in the trial.

Drugs 147

Drugs FDA Approval Clinical Trials Clinical Trials 147

Pharmaceutical Technology

APRIL 18, 2023

This news follows the treatment’s FDA approval on 10 March, making it the first-approved Rett Syndrome treatment in the US. The FDA approved the drug based on positive Phase III data from the Lavender trial. Acadia is also studying Daybue in Phase II clinical trials for Fragile X syndrome.

Drugs 255

Drugs FDA Approval Clinical Trials Clinical Trials 255

Pharmaceutical Technology

AUGUST 23, 2022

It comprises messenger ribonucleic acid (mRNA) that encodes for the initial spike protein of SARS-CoV-2 along with mRNA that encodes for Omicron BA.4/BA.5 5 variant spike protein. According to pre-clinical findings, a booster dose of this vaccine elicited a robust neutralising antibody response against the variants Omicron BA.1,

Vaccine 130

Vaccine Vaccination Gene Therapy Protein 130

Pfizer

JULY 26, 2022

Pfizer and BioNTech Advance COVID-19 Vaccine Strategy With Study Start of Next-Generation Vaccine Candidate Based on Enhanced Spike Protein Design. Pfizer and BioNTech Advance COVID-19 Vaccine Strategy With Study Start of Next-Generation Vaccine Candidate Based on Enhanced Spike Protein Design. deliesschef. Tue, 07/26/2022 - 17:41.

Vaccine 111

Vaccine Vaccination Protein Immune Response 111

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

DECEMBER 27, 2022

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

XTalks

JANUARY 6, 2025

Just before the end of 2024, Novo Nordisk nabbed US Food and Drug Administration (FDA) approval for its awaited hemophilia drug Alhemo (concizumab-mtci). In a press release announcing the approval, Novo said that Alhemo is the first subcutaneous injection of its kind in this patient population.

FDA Approval 105

FDA Approval Antibody Drugs Clinical Trials 105

XTalks

OCTOBER 23, 2024

Otsuka Pharmaceutical has announced positive interim results from its Phase III clinical trial for sibeprenlimab. The primary endpoint of the trial was a reduction in 24-hour urine protein-to-creatinine ratio (uPCR) compared to placebo after nine months of treatment, which indicates improved kidney function.

Trials 101

Trials Clinical Trials Clinical Trials Antibody 101

XTalks

AUGUST 17, 2020

As DMD is caused by a lack of functional dystrophin protein, which supports and strengthens muscle fibers, therapies designed to treat this rare genetic disorder have focused on exon skipping to avoid the area of the dystrophin gene that is mutated and produce a nearly full-length dystrophin protein.

Drugs 105

Drugs Clinical Trials Clinical Trials Gene 105

pharmaphorum

MAY 13, 2021

The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries. The trial is expected to enrol 99 ambulatory male patients, aged four to seven.

Gene Therapy 102

Gene Therapy Gene Trials Protein 102

XTalks

JULY 7, 2023

Pfizer’s alopecia JAK inhibitor Litfulo (ritlecitinib) has become its fourth US Food and Drug Administration (FDA) approved product in the past several weeks. Litfulo is entering the alopecia market to rival Eli Lilly’s Olumiant (baricitinib), which was approved as the first drug for alopecia areata a year ago.

Marketing 98

Marketing FDA Approval Trials Drugs 98

pharmaphorum

MARCH 10, 2021

The AI uses machine learning to measure what happens to human brain neural cells when treated with a drug, and could be “a more rapid and less expensive option” than clinical trials of novel therapeutics, according to the researchers.

Clinical Trials 94

Clinical Trials Clinical Trials Trials Drugs 94

Pharmaceutical Technology

MAY 3, 2023

The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). In January 2023, Tenaya announced FDA clearance for its Investigational New Drug (IND) application for TN-201.

Gene Therapy 242

Gene Therapy Gene Protein Trials 242

XTalks

NOVEMBER 1, 2024

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. billion in the US and $4.1 billion worldwide last year.

FDA Approval 85

FDA Approval Drugs Botox Sales 85

The Pharma Data

JANUARY 31, 2021

The phase 1 clinical trial was a randomized, double-blind and placebo-controlled study in 150 adult and elderly participants. The phase 1 clinical trial was a randomized, double-blind and placebo-controlled study in 150 adult and elderly participants. . CHENGDU, China , Feb. S-Trimer is intended to be adjuvanted.

Clinical Trials 52

Clinical Trials Clinical Trials Vaccine Vaccination 52

XTalks

MAY 15, 2023

Chiesi Global Rare Diseases in collaboration with Protalix BioTherapeutics announced the US Food and Drug Administration (FDA) approval of their new medication Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of Fabry disease. The initial half-life of Elfabrio was determined to be 78.9 ± 10.3

FDA Approval 98

FDA Approval Gene Genetics Clinical Trials 98

XTalks

JULY 13, 2023

It measures the phosphorylated form of the tau 181 protein isoform, a biomarker for Alzheimer’s disease pathology. The protein is found at low levels in the blood, necessitating high analytical sensitivity to measure it reliably.

Protein 98

Protein FDA Approval Development Clinical Trials 98