Pharmaceutical Technology

JUNE 8, 2023

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A). CMT1A is a progressive neuromuscular autosomal-dominant disease that leads to life-long loss of muscle function, as well as disability.

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European Pharmaceutical Review

DECEMBER 18, 2023

After eight weeks of treatment, patients’ depression severity scores dropped by an average of 19.1 As an oncologist for many years, I experienced the frustration of not being able to provide cancer care that treats the whole person, not just the tumour,” commented Manish Agrawal, MD, of Sunstone Therapies and lead author of the study.

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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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European Pharmaceutical Review

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has approved bluebird bio’s Zynteglo (betibeglogene autotemcel; beti-cel), the first cell-based gene therapy for the treatment of adult and paediatric patients with beta-thalassemia who require regular red blood cell transfusions. .

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European Pharmaceutical Review

NOVEMBER 4, 2022

The US-based researchers modelled personalised treatments for each virtual patient, assessing US Food and Drug Administration-approved aducanumab and donanemab, a therapy currently being evaluated and other potential AD therapies. These are identical to doses used in human trials for FDA approval.

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Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. g/dL and 45.4%

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Pharmaceutical Technology

APRIL 18, 2023

IntelGenx Corp announced that the US Food and Drug Administration (FDA) has approved the company’s Rizafilm VersaFilm new drug application (NDA) for the treatment of acute migraine. It dissolves rapidly releasing the active ingredient in the mouth, meaning there is no requirement for the patient to swallow a pill.

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Pharmaceutical Technology

JUNE 6, 2023

Findings from a two-year placebo-controlled, double-blind Phase II trial showed that Stargardt patients treated with gildeuretinol once a day as a pill experienced a statistically and clinically meaningful slowing of retinal damage over placebo. Alkeus Pharmaceuticals intends to submit a new drug application for gildeuretinol in 2024.

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pharmaphorum

SEPTEMBER 29, 2022

Regulators and health technology assessment (HTA) bodies are increasingly demanding the patient voice play a central role in drug development – making it riskier for developers to ignore the need for engagement than to embrace it. The patient centricity movement has been building momentum in recent years. Building relevance.

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pharmaphorum

NOVEMBER 2, 2022

In many ways, medicine has advanced rapidly and in myriad ways over the last few decades, and yet the opioid epidemic in the US proved that the challenge of helping patients in pain remains a difficult one to address. This leads to psychological harm becoming a significant factor in chronic pain, alongside the physical symptoms.

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European Pharmaceutical Review

AUGUST 23, 2022

The researchers studied 254 VA patients who were diagnosed with PTSD and hepatitis C between October 1999 and September 2019. Patients’ symptoms were monitored for PTSD and hepatitis C virus between two clinical visits over eight to 12 weeks. MDMA-assisted therapy successful in PTSD patients.

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pharmaphorum

JUNE 27, 2022

7008), which allows manufacturers to share vital information with healthcare payers and plans while treatments are pending Food and Drug Administration (FDA) approval, was passed by the House of Representatives and is awaiting approval in the Senate. Affecting patient care. The AMCP backed bill (H.R.

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Pharmaceutical Technology

OCTOBER 7, 2022

Provention Bio and Sanofi US have signed a co-promotion agreement to launch the former’s lead investigational drug candidate, teplizumab for the delay in the onset of clinical type 1 diabetes (T1D). The US Food and Drug Administration (FDA) is presently reviewing teplizumab for the delay of clinical T1D in people who are at risk.

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European Pharmaceutical Review

MAY 12, 2023

Innovative therapies for rare diseases The acquisition will help to drive the growth of CTI’s lead product, kinase inhibitor VONJO ® (pacritinib) “in treating myeloproliferative disease,” stated Dr Adam Craig, President, Chief Executive Officer and Interim Chief Medical Officer of CTI BioPharma.

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Legacy MEDSearch

OCTOBER 25, 2022

EndoStim , a medical device company developing and commercializing a first-in-class implantable neurostimulation treatment for drug refractory gastroesophageal reflux disease (GERD), announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Device Designation for the Company’s EndoStim System.

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European Pharmaceutical Review

NOVEMBER 28, 2023

The US Food and Drug Administration (FDA) has approved the first drug for desmoid tumours (desmoid fibromatosis), an oral gamma secretase inhibitor. According to the FDA, this clinical trial evaluated 142 adult patients with progressing desmoid tumours not suitable to surgery.

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Legacy MEDSearch

DECEMBER 8, 2022

took a step towards its vision of transforming chronic care management with Polso , the company’s Remote Patient Monitoring (RPM) solution, achieving 510(k) clearance from the U.S. Food and Drug Administration (FDA). ChroniSense Medical Ltd. and globally. About ChroniSense Medical. About Rainbow Medical.

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European Pharmaceutical Review

MARCH 18, 2024

A Phase III trial has shown that compared oral drug delivery, administering levodopa through an infusion pump led to nearly two hours of day (1.72) of additional time in which the medicine reduced symptoms in Parkinson’s patients. There were 381 Parkinson’s patients enrolled in the trial. and Joan A.

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European Pharmaceutical Review

DECEMBER 5, 2023

Efficacy of the treatment was compared to placebo in patients with lower risk myelodysplastic syndromes (MDS) relapsed/refractory or ineligible for erythropoiesis stimulating agents (ESAs). The promise of telomerase inhibitors for treating blood cancer Trial results The clinical trial enrolled a total of 178 patients. g/dL for placebo.

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Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). In the study, Hemgenix reduced or eliminated the need for prophylactic treatment in 94% of patients. Hemgenix is the first and only gene therapy approved for haemophilia B.

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European Pharmaceutical Review

APRIL 29, 2024

The US Food and Drug Administration (FDA) has approved the adeno-associated virus (AAV)-based gene therapy BEQVEZ (fidanacogene elaparvovec-dzkt) for certain adults with haemophilia B. This authorisation of BEQVEZ is based on results from the pivotal Phase III BENEGENE-2 study.

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European Pharmaceutical Review

DECEMBER 1, 2023

During CPHI Barcelona, EPR Editor Caroline Peachey asked Anil Kane, Global Head of Technical & Scientific Affairs, Pharma Services, at Thermo Fisher Scientific about the dynamic landscape of pharmaceutical drug development. What are the trends affecting drug development today? Prefilled syringes bring a range of benefits.

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Pharmaceutical Technology

JUNE 8, 2023

The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).

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European Pharmaceutical Review

JULY 4, 2023

As drug development professionals know, global health crises like the COVID-19 pandemic provide the public with valuable insights into how clinical research and regulatory processes work. What are Antibody Drug Conjugates and Multispecific Antibody Therapeutics?

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European Pharmaceutical Review

JANUARY 3, 2023

A marketing authorisation application (MAA) has been submitted by The Janssen Pharmaceutical Companies of Johnson & Johnson to the European Medicines Agency (EMA) for approval of talquetamab, a bispecific t-cell engager antibody for the treatment of patients with relapsed or refractory multiple myeloma (RRMM).

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Pharmaceutical Technology

JANUARY 19, 2023

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

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European Pharmaceutical Review

JANUARY 5, 2024

“ Treatment with evenamide as an add-on to antipsychotics in treatment-resistant schizophrenia (TRS) patients has produced benefits that have never been reported before,” Ravi Anand, Newron’s Chief Medical Officer shared. This is compared to daily dosing, therefore helping to reduce the peak to trough variation patients can experience.

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Pharmaceutical Technology

APRIL 3, 2023

It also decreases the number of injections needed by patients who require a dose of 80 mg/mL or above. Sandoz Europe Region head Rebecca Guntern said: “Living with a chronic disease can take a significant toll on a patient’s quality of life.

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European Pharmaceutical Review

JANUARY 25, 2023

The edited cells, exa-cel, are then infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT). The elevation of HbF by exa-cel has the potential to reduce or eliminate painful and debilitating vaso-occlusive crises for patients with SCD and alleviate transfusion requirements for patients with TDT.

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European Pharmaceutical Review

JULY 28, 2023

Publication of results of the Phase III SKYLARK study “is a pinnacle moment in treating postpartum depression ( PDD ),” stated Dr Kristina M Deligiannidis, Professor at the Institute of Behavioral Science at the Feinstein Institutes, the trial’s principal investigator, lead author of the paper.

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Pharmaceutical Technology

JANUARY 16, 2023

In October last year, CARsgen submitted a new drug application (NDA) for CT053 to China’s National Medical Products Administration (NMPA). The US Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations to the company’s zevor-cel in 2019.

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Pharma Marketing Network

MARCH 11, 2025

Which platforms provide valuable insights for reaching healthcare professionals (HCPs) and patients? Digital platforms provide a way to streamline outreach efforts, measure campaign performance, and engage with both healthcare professionals and patients.

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European Pharmaceutical Review

FEBRUARY 7, 2023

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, an adeno-associated virus (AAV)-based gene therapy for Danon disease. “RP-A501 Ultimately, this leads to heart failure and can be fatal for male patients.

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Pharmaceutical Technology

NOVEMBER 7, 2022

A lead product candidate of the company, SNK01 is presently being analysed as a single agent and along with other agents, including checkpoint inhibitors and cell engagers, in trials to treat advanced refractory solid tumours. . “We Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

NOVEMBER 23, 2022

The US Food and Drug Administration (FDA) has granted approval for CSL Behring’s adeno-associated virus vector-based gene therapy, Hemgenix (etranacogene dezaparvovec), to treat haemophilia B (congenital Factor IX deficiency) in adult patients. at six months and 24 months, respectively, following infusion.

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European Pharmaceutical Review

MAY 24, 2023

The US Food and Drug Administration (FDA) has approved the first-ever redosable gene therapy for the rare skin disease dystrophic epidermolysis bullosa (DEB). VYJUVEK (beremagene geperpavec-svdt) is authorised for DEB patients six months of age or older.

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European Pharmaceutical Review

OCTOBER 2, 2023

has discontinued the Phase IIb clinical trial of its lead antibiotic candidate, ibezapolstat, for Clostridioides difficile infection (CDI). In the finished Phase IIa segment of this trial, 10 patients with diarrhoea caused by C. Every patient was investigated for recurrence for 28± 2 days. Developing a front-line C.

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