XTalks

DECEMBER 12, 2023

After receiving US Food and Drug Administration (FDA) approval for Fabhalta (iptacopan) last week for the treatment of the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), Novartis presented trial data yesterday showing the drug’s promise in another indication.

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Pharmaceutical Technology

SEPTEMBER 5, 2022

The US Food and Drug Administration (FDA) has approved the expanded usage of Vertex Pharmaceuticals ’ Orkambi (lumacaftor/ivacaftor) to treat cystic fibrosis (CF) in children aged 12 months to under 24 months. It enrolled 46 children aged one to under two years with the F/F genotype.

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Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

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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. The study is one of the largest DMD Phase III trials to date.

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Pharmaceutical Technology

MAY 10, 2023

The US Food and Drug Administration (FDA) has granted approval for AstraZeneca ’s Farxiga (dapagliflozin) to reduce the risks of cardiovascular (CV) death, hospitalisation for heart failure (hHF) and urgent heart failure (HF) emergency care visits in HF adult patients.

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Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. As an FDA-approved oral drug, the side-effects of cladribine are quite manageable and it has been well evaluated for pharmacokinetics.

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pharmaphorum

JANUARY 26, 2022

Immunocore has secured a piece of biotech industry history, becoming the first company to get an FDA approval for a cancer therapeutic based on T cell receptor (TCR) technology. The post Immunocore claims first-ever FDA approval for TCR cancer therapy appeared first on.

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pharmaphorum

AUGUST 4, 2021

Bristol-Myers Squibb’s HDAC inhibitor Istodax has been on the US market for a decade as a treatment for peripheral T-cell lymphoma (PTCL), but will now be withdrawn from sale after a failed phase 3 trial. The post Failed trial nixes another FDA approval, this time for BMS’ Istodax appeared first on.

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XTalks

OCTOBER 27, 2022

The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the US Food and Drug Administration (FDA) approved Tecvayli (teclistamab-cqyv) for the treatment of relapsed or refractory multiple myeloma. The FDA approval of Tecvayli makes it the first bispecific BCMA-directed CD3 T-cell engager approved in the US.

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Drug Discovery World

AUGUST 21, 2023

Ipsen’s Sohonos (palovarotene) has become the first drug approved for patients with fibrodysplasia ossificans progressiva (FOP) in the US, following the granting of a marketing authorisation by the Food and Drug Administration (FDA). The EU’s regulator chose not to recommend the drug in January 2023.

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XTalks

JANUARY 25, 2023

Massachusetts-based TheracosBio has received US Food and Drug Administration (FDA) approval for diabetes med Brenzavvy (bexagliflozin) to help improve glycemic control in adults with type 2 diabetes. It is the first FDA-approved SGLT2 inhibitor for any animal species. What is the Animal Drug Regulatory Process?

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XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

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pharmaphorum

JANUARY 16, 2023

China’s Luye Pharma has claimed its first FDA approval, getting a green light for Rykindo, an injectable formulation of the antipsychotic risperidone given every two weeks. ” The post Luye claims its first FDA approval, for bi-weekly schizophrenia drug appeared first on.

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pharmaphorum

SEPTEMBER 16, 2021

Takeda has had a run of bad luck with its Wave1 pipeline of new drug candidates of late, but can now celebrate a vestry after getting FDA approval for first-in-class lung cancer therapy Exkivity. The post Takeda gets a win for its Wave1 pipeline, as Exkivity nabs FDA approval appeared first on.

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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.

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XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

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XTalks

MARCH 10, 2023

But with Skyclarys (omaveloxolone), the first and only drug to treat Friedreich’s ataxia in patients aged 16 years and older, Reata Pharmaceuticals has developed a new treatment option to improve neurological function. This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher.

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XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s s candidate drug AMX0035 (sodium phenylbutyrate/taurursodiol) for the treatment of amyotrophic lateral sclerosis, or ALS. This time, the group is backing a drug that they believe will greatly benefit ALS patients.

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XTalks

JUNE 2, 2021

The US Food and Drug Administration (FDA) has granted approval to Amgen’s KRAS inhibitor Lumakras (sotorasib) as the first treatment for non-small cell lung cancer (NSCLC) harboring the KRAS G12C mutation. The sotorasib approval is therefore a major breakthrough in the world of cancer and targeted therapies.

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Pharmaceutical Technology

MAY 5, 2023

The US Food and Drug Administration (FDA) has accepted Optinose’s supplemental new drug application (sNDA) for Xhance (fluticasone propionate) to treat chronic rhinosinusitis, which is now under review. The regulator has set 16 December 2023 as a prescription drug user fee act (PDUFA) target goal date.

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XTalks

DECEMBER 2, 2022

Swiss company Ferring Pharmaceuticals has won a historic approval from the US Food and Drug Administration (FDA) for its fecal transplant-based therapy Rebyota (fecal microbiota, live-jslm) to reduce the recurrence of bacterial infection. The therapy is the first of its kind to be approved in the US. Difficile. “As

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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).

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XTalks

MAY 31, 2024

Allecra Therapeutics, a clinical-stage biopharma company focused on combating antibiotic resistance, received approval from the US Food and Drug Administration (FDA) for Exblifep (cefepime/enmetazobactam). How Does Exblifep Work? In a study with cUTI patients, 72.6

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XTalks

NOVEMBER 1, 2022

Rinvoq received its sixth FDA approval, this time it is for treating adults with non-radiographic axial spondyloarthritis, a form of inflammatory arthritis that affects the spine. JAK inhibitors block cell signaling that cause inflammation, providing patients an alternative method to regulating an overactive immune system.

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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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pharmaphorum

MAY 5, 2022

Three years after it was formed as a spinout of Japanese drugmaker Takeda, Phathom Pharmaceuticals has racked up its first FDA approvals, and claimed a sizeable $260 million in new financing to help with the rollout. The post Takeda spinout Phathom claims its first FDA approvals appeared first on.

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Pharmaceutical Technology

MAY 16, 2023

Health Canada has accepted a new drug submission (NDS) for Eisai and Biogen ’s lecanemabto to treat early Alzheimer’s disease (AD). In January 2023, the US Food and Drug Administration (FDA) approved lecanemab under the accelerated approval pathway to treat AD.

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pharmaphorum

AUGUST 13, 2020

Exon-skipping drugs are used to patch the mutations and allow the gene to produce partially functional dystrophin. Viltepso can be used to treat around 8% of DMD patients and previously approved DMD drugs has not been shown to change the course of the disease.

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XTalks

SEPTEMBER 16, 2022

Last week, Bristol Myers Squibb (BMS) announced that their therapy Sotyktu (deucravacitinib) was approved by the US Food and Drug Administration (FDA) as a first-line treatment for adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. Sotyktu (deucravacitinib) Clinical Results.

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Pharmaceutical Technology

AUGUST 16, 2022

According to the deal, Horizon will provide the funding to develop the therapy until the conclusion of its two Phase II clinical trials. Under the agreement, Horizon will hold an option for the acquisition of the ADX-914 programme, which is exercisable through a period after concluding the Phase II trials.

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Pharmaceutical Technology

JUNE 9, 2023

Rocket Pharmaceuticals has received fast track and orphan drug designations for RP-A601 from the US Food and Drug Administration (FDA) to treat plakophilin-2-related arrhythmogenic cardiomyopathy (PKP2-ACM). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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pharmaphorum

MARCH 9, 2022

US House Energy & Commerce Committee Chair Frank Pallone (D-NJ) has introduced a new bill to enhance the Food and Drug Administration’s (FDA) authority to ensure products that receive accelerated approval are providing a clinical benefit to patients. Covis Pharma’s Makena, has been a notable example of this.

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XTalks

JANUARY 28, 2022

Kimmtrak has also become the first bispecific T cell engager to be FDA-approved for the treatment of a solid tumor. Immunocore received Breakthrough Device Designation for Kimmtrak from the FDA in February 2021 followed by acceptance of its Biologics License Application (BLA) several months later by both US and European regulators.

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XTalks

FEBRUARY 8, 2024

The top 15 diabetes drugs in 2023, according to 2022 sales data, reflect the current state of diabetes management and hint at the evolving needs and trends within this critical area of healthcare. Read on to learn more about the top 15 diabetes drugs in 2023, based on 2022 sales statistics. mL subcutaneous solution (2 mg/1.5

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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. Xtalks interviewed Dr. Neilan to learn about his work at NORD and how NORD aims to enhance patient access in rare disease clinical trials.

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XTalks

APRIL 6, 2023

Mounjaro (tirzepatide) is a dual glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) agonist, which makes it the first-in-class drug that activates both receptors to help regulate blood sugar and appetite. A digital campaign for the drug was launched in January. The “What If?”

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pharmaphorum

AUGUST 16, 2021

Merck & Co has extended its range of cancer medicines in the US after claiming FDA approval for Welireg – a drug it acquired as part of its $2.2 This first FDA approval triggers a $50 million payment to former Peloton shareholders, with another $50 million due on commercial launch. Merck paid $1.05

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