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. There are three more drug candidates with major trial readouts that are expected in 2023.

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European Pharmaceutical Review

JANUARY 10, 2023

Clarivate Plc has released its Drugs to Watch 2023 report — among 70 of the drugs highlighted, including potential blockbuster drugs, the majority were revealed to be personalised medicines. The report offers predictive analysis of drugs entering the market or launching key indications in 2023.

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Food and Drug Administration FDA Food Medicine 87

pharmaphorum

DECEMBER 22, 2022

The life sciences industry is flexing towards innovation in new areas, faster than ever before, and increasing patient care in astonishing ways. We can now measure patient activity, steps, and movement continuously and in real time, which serves as a new potential indicator of treatment effectiveness.

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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. Novartis and Incyte Corp’s Jakafi was the first FDA-approved drug for the treatment of myelofibrosis in November 2011.

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Pharmaceutical Technology

MAY 2, 2023

The US Food and Drug Administration (FDA) has accepted the supplemental biologics licence application submitted by Bristol Myers Squibb for Reblozyl (luspatercept-aamt) as a first-line treatment of anaemia in adults with lower-risk myelodysplastic syndromes (MDS).

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Food and Drug Administration FDA Safety Prescription 98

pharmaphorum

DECEMBER 19, 2022

The US Food and Drug Administration (FDA) has approved Swiss drugmaker Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firedenovec-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 tumours.

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Pharmaceutical Technology

JUNE 1, 2023

The US Food and Drug Administration (FDA) has granted a combination of AstraZeneca and MSD ’s Lynparza (olaparib), with standard therapies for treating BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC). Safety and tolerability were in line with that observed in prior trials and the known profiles of the medicines.

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Food and Drug Administration FDA Food Safety 97

PM360

OCTOBER 13, 2023

HCPs need pharma, and pharma needs HCPs—so let’s make sure that this next chapter of engagement is one of partnership, in pursuit of the interest of patients.” Only 15% of prescribers think the drugs come at reasonable cost to patients or have decent insurance coverage eligibility. Three quarters of U.S.

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Pharmaceutical Technology

MAY 18, 2023

The US Food and Drug Administration (FDA) has accepted Ardelyx’s resubmission of a new drug application (NDA) for XPHOZAH (tenapanor) to control serum phosphate in adults with chronic kidney disease on dialysis who have had insufficient response or intolerance to a phosphate binder treatment.

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Pharmaceutical Technology

MAY 31, 2023

The US Food and Drug Administration (FDA) has accepted Bristol Myers Squibb’s new drug application (NDA) for repotrectinib for priority review. Repotrectinib is a tyrosine kinase inhibitor (TKI) intended to treat patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC).

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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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Pharmaceutical Technology

MAY 25, 2023

Celltrion USA has received approval from the US Food and Drug Administration (FDA) for Humira (adalimumab) biosimilar, Yuflyma (adalimumab-aaty) , for multiple indications. It will be offered to patients in prefilled syringe and autoinjector administration options. The company intends to launch the biosimilar in the US in July 2023.

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Pharmaceutical Technology

MAY 11, 2023

The regulator accepted and gr anted priority review for the sNDA in January 2023. Otsuka Pharmaceutical representative director and president Makoto Inoue stated: “Today marks a major milestone for patients, caregivers and families navigating the complexities of agitation associated with dementia due to Alzheimer’s disease.

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Legacy MEDSearch

JANUARY 13, 2023

Hip Innovation Technology , LLC (HIT), a medical device company developing innovative orthopaedic device solutions to advance the quality of life and quality of care for patients, is pleased to announce the first U.S. Safety will be assessed through the collection of device-related adverse events and patient quality of life metrics.

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Pharma Leaders

MAY 18, 2023

The US Food and Drug Administration (FDA) has accepted Ardelyx’s resubmitted New Drug Application (NDA) for XPHOZAH (tenapanor) to control serum phosphate in adult patients with chronic kidney disease on dialysis who have had an insufficient response or intolerance to a phosphate binder therapy.

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Pharmaceutical Technology

MAY 26, 2023

After earning multimillion dollar revenues while being an authorised preferred treatment for Covid-19, the US Food and Drug Administration (FDA) has granted a full approval to Pfizer’s oral antiviral Paxlovid (nirmatrelvir + ritonavir). However, other studies found that the relapse in symptoms occurs in patients independent of Paxlovid use.

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Food and Drug Administration FDA Side effects Safety 59

Pharmaceutical Technology

JUNE 1, 2023

Additionally, when asked whether the FDA should consider a potential accelerated approval, panellists voted 15 to one against that regulatory pathway for the drug. Additionally, when asked whether the FDA should consider a potential accelerated approval, panellists voted 15 to one against that regulatory pathway for the drug.

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European Pharmaceutical Review

FEBRUARY 21, 2023

SYFOVRE (pegcetacoplan injection) is the first and only treatment approved by the US Food and Drug Administration (FDA) for geographic atrophy (GA), a leading cause of blindness. SYFOVRE is approved for GA patients with or without subfoveal involvement and provides dosing flexibility via a dosing regimen of every 25 to 60 days.

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PM360

APRIL 7, 2023

Patient Pages: How Healthcare Costs Affect Insured Patients Today A new study shows an insured American with an employer-sponsored health insurance plan can expect to spend more than $320,000 (including insurance premiums and out-of-pocket costs) during his/her adult lifetime.

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Pharmaceutical Technology

APRIL 14, 2023

The US Food and Drug Administration (FDA) has rejected Eli Lilly’s biologic licence application (BLA) for the ulcerative colitis (UC) drug mirikizumab over manufacturing concerns. No concerns related to the clinical data package, safety or the medicine label. The regulator has issued a complete response letter.

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Pharmaceutical Technology

APRIL 19, 2023

The US Food and Drug Administration (FDA) has accepted Arcutis Biotherapeutics ’ new drug application (NDA) for roflumilast foam 0.3% to treat patients aged nine years and above with seborrheic dermatitis. in patients with seborrheic dermatitis. in patients with seborrheic dermatitis.

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Pharmaceutical Technology

JUNE 14, 2023

Ipsen has received US Food and Drug Administration (FDA) approval for Bylvay (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome (ALGS). More than 90% of patients who received treatment with odevixibat were pruritus responders.

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pharmaphorum

SEPTEMBER 30, 2022

Patient organisations have been celebrating the FDA approval yesterday of Amylyx’ amyotrophic lateral sclerosis (ALS) therapy Relyvrio, after not one but two advisory committee meetings that arrived at different conclusions about the drug. An oral version of edaravone was cleared this year.

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Pharmaceutical Technology

NOVEMBER 1, 2022

The US Food and Drug Administration (FDA) has granted Fast Track designation for Dyne Therapeutics’ DYNE-251 to treat Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. In non-human primates, the product candidate showed a good safety profile.

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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). In April 2023, the FDA approved the investigational new drug (IND) application for RP-A601 to begin a Phase I clinical trial.

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Pharmaceutical Technology

APRIL 3, 2023

Tango Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application for TNG260 to treat STK11-mutant cancers. We look forward to initiating the Phase 1/2 clinical trial of TNG260 in the second half of 2023.”

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Food and Drug Administration FDA Food Safety 52

European Pharmaceutical Review

SEPTEMBER 18, 2023

The first and only treatment for anaemic patients with myelofibrosis has been approved by the US Food and Drug Administration (FDA). While transfusions are often required, more than 30 percent of patients will discontinue treatment due to anaemia.​

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European Pharmaceutical Review

MARCH 4, 2024

Indeed, in 2022, biologics constituted 40 percent of all US Food and Drug Administration (FDA) approved drugs, projecting a compound annual growth rate (CAGR) of 9.5 The emergency use approval from the FDA of lipid nanoparticle- (LNP-) based mRNA vaccines during the COVID-19 pandemic attested the significance of LNPs.

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European Pharmaceutical Review

JUNE 27, 2023

AC Immune SA has received Fast Track designation from the US Food and Drug Administration (FDA) for its anti-amyloid beta (Abeta) active immunotherapy vaccine candidate for Alzheimer’s disease. The anti-Abeta therapy “specifically targets the most toxic forms of Abeta” according to Dr Andrea Pfeifer, CEO of AC Immune SA.

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European Pharmaceutical Review

MAY 7, 2024

A novel intravesical targeted drug delivery system for patients with bladder cancer has demonstrated positive data in updated results from a Phase I trial. Johnson & Johnson’s Phase I study is assessing the safety and efficacy of TAR-210 as a delivery method for sustained, local release of erdafitinib into the bladder.

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Food and Drug Administration Food Patients Safety 76

European Pharmaceutical Review

OCTOBER 21, 2022

Clinical trial results presented at the US IDWeek conference, revealed a three-dose series of the HEPLISAV-B vaccine prevents hepatitis B virus (HBV) in HIV patients not previously vaccinated against or infected with the virus. At week 28, the study assessed the vaccine’s safety and levels of anti-HBV surface antibodies (HBsAbs).

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Pharmaceutical Technology

FEBRUARY 8, 2023

The US Food and Drug Administration (FDA) has accepted Aldeyra Therapeutics’ new drug application (NDA) for topical ocular reproxalap to treat signs and symptoms of dry eye disease. The regulator has assigned 23 November 2023 as the Prescription Drug User Fee Act (PDUFAs) date for its decision.

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European Pharmaceutical Review

JANUARY 9, 2024

TALZENNA the first and only PARP inhibitor licensed in the EU for use with XTANDI for patients with mCRPC, with or without gene mutations” The treatment is indicated for adults with metastatic castration-resistant prostate cancer (mCRPC) in individuals where chemotherapy is not clinically indicated.

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Food and Drug Administration Safety Food Patients 83

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

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FDA Safety Patients Doctors 101

PM360

DECEMBER 14, 2023

A New Online Destination for HCPs HealthCentral Corporation , operator of leading digital platforms that serve patients living with chronic illness, their care partners, and health professionals, has announced the newest addition to its suite of HCP support sites: MedCentral.

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European Pharmaceutical Review

APRIL 16, 2024

Novartis has released new data from the first and only Phase III study to demonstrate significant proteinuria reduction by targeting the complement system in patients with the rare kidney disease IgA nephropathy (IgAN). Fabhalta was well tolerated with a favorable safety profile consistent with previously reported data, Novartis confirmed.

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Pharmaceutical Technology

APRIL 26, 2023

The US Food and Drug Administration (FDA) has granted orphan drug designation to IN8bio’s INB-400 and INB-410 to treat a range of malignant gliomas, including newly diagnosed glioblastoma multiforme (GBM). Participant enrolment is anticipated to commence in the second half of 2023.

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