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 RMAT designation will provide the benefits of added intensive FDA guidance and expedited review through the programme’s development.

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pharmaphorum

DECEMBER 22, 2022

Prediction 1: the FDA will become more directive and collaborative on guidelines for DCTs. This will lead to more pre-protocol design discussions between sponsors and CROs and regulatory bodies seeking input on new and potentially acceptable approaches to, for example, measure digital endpoints.

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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. Yuflyma represents the company’s fifth biosimilar and second anti-TNF biosimilar to receive US FDA approval.

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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

JANUARY 22, 2023

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. More therapies to meet unmet needs GlobalData predicts that at least 35 US regulatory decisions on drugs for rare diseases are on the horizon for 2023. GlobalData is the parent company of Pharmaceutical Technology.

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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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Legacy MEDSearch

JANUARY 13, 2023

implantation of the HIT Reverse Hip Replacement System (Reverse HRS), under an FDA approved Investigational Device Exemption (IDE). The IDE Study is being conducted to determine the safety and effectiveness of the HIT Reverse HRS in Primary Total Hip Arthroplasty (THA). About Hip Innovation Technology, LLC.

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European Pharmaceutical Review

FEBRUARY 2, 2024

Biogen’s decision A strategic review of the company’s R&D work evaluated the time and investment required for the post-marketing confirmatory ENVISION study and the likely advancements in the field by the time of potential ADUHELM FDA traditional approval, Biogen explained.

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PM360

APRIL 7, 2023

Furthermore, many people underestimate their healthcare costs and do not save enough for annual expenditures, often leading to delays in treatment that can be detrimental to the patient. CMS factored in the efficacy of the device as well as its safety beyond the use of opioids. Fitzgerald, CEO and President, Apollo Intelligence.

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pharmaphorum

DECEMBER 8, 2022

The FDA has started its review of Pfizer’s respiratory syncytial virus (RSV) vaccine for older adults, fast-tracking the biologics license application (BLA) with a decision due in May 2023. The post RSV vaccine race in balance as FDA sets May date for Pfizer decision appeared first on.

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pharmaphorum

NOVEMBER 16, 2022

has announced that its platinum-resistant ovarian cancer drug ELAHERE (mirvetuximab soravtansine-gynx) has been granted FDA accelerated approval. RxDx Assay – Roche’s immunohistochemistry (IHC) companion diagnostic for identifying ovarian patients eligible for ELAHERE – has been contemporaneously approved by the FDA. ImmunoGen, Inc.

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FDA Patients Safety Medicine 67

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

OCTOBER 30, 2023

According to Atamyo Therapeutics, the condition is characterised by progressive muscular weakness, leading to loss of the ability to walk independently. Moreover, no unexpected safety signal for the gene therapy was identified in the LGMD2I/R9 study. Individuals with the disease are also prone to respiratory impairment.

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European Pharmaceutical Review

OCTOBER 24, 2023

PENBRAYA (meningococcal groups A, B, C, W and Y vaccine), the first and only pentavalent vaccine that provides the broadest serogroup coverage of any meningococcal vaccine available in the US for meningococcal disease in individuals aged 10 to 25 years old, has been approved by the US Food and Drug Administration (FDA). About PENBRAYA?

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European Pharmaceutical Review

DECEMBER 22, 2023

This represents a 53 percent premium on Karuna’s closing stock price on 21 December 2023. The deal includes Karuna’s lead asset KarXT (xanomeline-trospium), a potential first-in-class treatment for schizophrenia. KarXT targets both the M1 and M4 muscarinic receptors, resulting in a differentiated safety and efficacy profile.

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Food and Drug Administration Side effects Safety Food 91

Pharmaceutical Technology

MARCH 20, 2023

Leading the race to commercialisation is Intercept Pharmaceuticals, which, in December 2022, resubmitted the new drug application (NDA) for its obeticholic acid (OCA), an FXR agonist, as a Class 2 resubmission (which has a six-month review period) for the treatment of patients with pre-cirrhotic liver fibrosis due to NASH.

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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 These actions occur at both the delivery site and other systemic locations, thereby promoting an immunogenic response that leads to cancer cell death.

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European Pharmaceutical Review

AUGUST 22, 2023

The US Food and Drug Administration (FDA)-approved treatment, is the first RSV vaccine indicated for infants from birth to six months of age. US FDA granted approval of Abrysvo to Pfizer Inc. This news follows similar regulatory action by the US FDA in recent months. percent within 90 days after birth.

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Food and Drug Administration FDA Government Food 77

European Pharmaceutical Review

MAY 8, 2024

Although we have yet to understand the human microbiome and its role in disease fully, the scientific evidence on the efficacy of complex live biotherapeutic products (LBPs) in modulating the microbiome is striking, as evidenced by the approval of the first two commercial donor-derived products, Rebyota and VOWST, in 2022 and 2023, respectively.

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pharmaphorum

JANUARY 5, 2023

The clock has started ticking on the FDA’s review of Sanofi and AstraZeneca’s one-shot antibody to protect newborns and infants from respiratory syncytial virus (RSV), with a decision due in the third quarter – just in time for the 2023/24 RSV season.

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European Pharmaceutical Review

MAY 15, 2023

HARMONIE investigated the efficacy and safety of a single intramuscular dose of nirsevimab. With the data having been collected in a real-world (RW) setting during the 2022-2023 RSV season, it was “gathered at a time when RSV was recovering from the dip in case numbers during COVID lockdown” Professor Openshaw added.

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Safety Leads Manufacturing Medicine 80

pharmaphorum

OCTOBER 27, 2022

The FDA has accepted for review Seres Therapeutics’ Biologics License Application (BLA) for SER-109, an investigational oral microbiome therapeutic for the prevention of recurrent Clostridium difficile infection (rCDI). If approved, SER-109 would be the first-ever FDA-approved oral microbiome therapeutic. Now at $6.79

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European Pharmaceutical Review

AUGUST 31, 2023

Overcoming challenges in patient safety, manufacturing and supply of radiopharmaceuticals As clinical trials progress and the first results are published, companies’ best candidates will emerge in the next five years. Hospital staff must also be protected from radiation while handling the agent. Through its longer half-life of 12.7

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Food and Drug Administration Manufacturing Safety Transportation 75

European Pharmaceutical Review

APRIL 10, 2024

The recent FDA approvals of two microbiome products in the US, in November 2022 and April 2023, 1-2 represent a significant milestone, maturing the industry and paving the way for advancement and regulatory clearance of additional microbiome products in broader indications.

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European Pharmaceutical Review

MAY 4, 2023

Data announced at the European Lung Cancer Congress (ELCC) earlier this month revealed the long-term efficacy and safety of RYBREVANT ® (amivantamab) in patients with post-platinum EGFR Ex20ins-mutated advanced non-small cell lung cancer (NSCLC). 4, 5 Why was the data presented at ELCC 2023 on the bispecific monoclonal antibody significant?

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Food and Drug Administration Safety Medicine Patients 88

Pharmaceutical Technology

APRIL 13, 2023

Lower DCT usage for Phase I studies may be accounted for due to the heavier focus on safety and efficacy, and increased monitoring of trial participants in-person. Metabolic disorders have remained the leading therapy area for DCT adoption for over five years, mainly due to the use of wearables, such as glucometers, and other smart devices.

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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

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pharmaphorum

AUGUST 18, 2022

Astellas remains on course to be the first drugmaker to bring a new non-hormonal therapy for symptoms of menopause to market, after the FDA started a review of its orally-active neurokinin-3 (NK3) receptor antagonist fezolinetant. billion yen ($97 million) charge as an R&D expense in its fiscal first quarter.

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European Pharmaceutical Review

MARCH 14, 2024

For IgA nephropathy (IgAN), key highlights include the US Food And Drug Administration (FDA) approval of Calliditas Therapeutics’ TARPEYO and the Committee for Medicinal Products for Human Use (CHMP)’s recommendation of a conditional marketing authorisation for Filspari (sparsentan). Internet] Vera Therapeutics. cited 2024Mar].

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Food and Drug Administration Patients Leads Side effects 91

European Pharmaceutical Review

MARCH 14, 2024

For IgA nephropathy (IgAN), key highlights include the US Food And Drug Administration (FDA) approval of Calliditas Therapeutics’ TARPEYO and the Committee for Medicinal Products for Human Use (CHMP)’s recommendation of a conditional marketing authorisation for Filspari (sparsentan). Internet] Vera Therapeutics. cited 2024Mar].

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Food and Drug Administration Patients Leads Side effects 90

European Pharmaceutical Review

FEBRUARY 13, 2023

How CAR T-cell therapy ide-cel works Dr Giralt explained that the CAR T-cell therapy recognises and binds to B-cell maturation antigens (BCMA) on the surface of MM cells, leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

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Food and Drug Administration Engineering Safety Patients 111

pharmaphorum

SEPTEMBER 14, 2022

SparingVision’s lead candidates are both targeting inherited retinal diseases (IRDs), a group which already has a gene therapy on the market in Novartis’ Luxturna (voretigene neparvovec), which was approved by the FDA in 2017 for biallelic RPE65 mutation-associated retinal dystrophy, a group which includes a small group of RP patients.

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Pharmaceutical Technology

JUNE 8, 2023

While the company previously shared interim biomarker and safety data from the Phase I/II AEROW trial (NCT05248230) in November 2022, the update features further results detailing the drug’s effects on quality-of-life and spirometry-measured outcomes in a limited set of patients.

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Pharmaceutical Technology

JUNE 16, 2023

Belgium-headquartered ANeuroTech is participating in a Series B round, which will raise up to $150m to power the pivotal programme of its lead candidate ANT01 for use as an adjunctive treatment for major depressive disorder (MDD). The company aims to run an interim analysis of the trial by mid-2024 at the very latest.

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Food and Drug Administration Side effects Safety Recruitment 52

Pharmaceutical Technology

MARCH 24, 2023

Amgen’s (US) Amjevita (adalimumab) is the first Humira biosimilar, launched in February 2023, and seven other FDA-approved biosimilars are expected to arrive on the US market later this year. In addition, two more Humira biosimilars are currently under FDA review and both are anticipated to launch in 2023.

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pharmaphorum

SEPTEMBER 21, 2022

The European Commission has followed the lead of the US FDA and approved AstraZeneca’s Tezspire as an add-on maintenance therapy for patients with severe asthma, becoming the first and only biologic that can be used in all patients, and not restricted to those with specific forms of the disease. billion or more.

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Sales Biopharma Medicine Patients 101

pharmaphorum

SEPTEMBER 2, 2022

The FDA has cleared the IL-36 receptor inhibitor under the Spevigo brand name to treat the devastating eruptions of painful blisters, known as flares, that are a characteristic of GPP. Meanwhile, spesolimab is also being tested to potentially treat other skin diseases, such as palmoplantar pustulosis and hidradenitis suppurativa.

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