Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. For manufacturing, it’s no different.

Therapies 98

Therapies Small Molecule Production Disease 98

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

Therapies 98

Therapies Clinical Trials DNA Trials 98

BioPharma Drive: Drug Pricing

JUNE 27, 2023

The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.

Therapies 108

Therapies Treatment Marketing 108

Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? How is the market demand for lentiviral vectors evolving, and what factors are influencing this growth? There is a very significant increased market demand for lentiviral vectors. New therapeutic areas are emerging in a powerful way.

Therapies 59

Therapies Vaccine Marketing Disease 59

ASPET

OCTOBER 20, 2023

However, Pramlintide an adjunct to insulin therapy, enhances glucagon suppression and thereby offers improved glycaemic control. Although clinical benefits of Pramlintide are well reported, there still exists a high patient resistance for the therapy as separate injections for Pramlintide and insulin are required to be administered.

Therapies 100

Therapies Pharmacokinetics Treatment Marketing 100

The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS in December 2022 about changes to their initial 2017 partnership whereby Daiichi Sankyo obtained the Marketing Authorization for Yescarta ®. to Gilead Sciences K.K.,

Therapies 40

Therapies Marketing Small Molecule Science 40

The Premier Consulting Blog

NOVEMBER 12, 2024

Recently, we convened a panel of national payer pharmacy decision-makers to ask them about how they evaluate 505(b)(2)s and whether an approved FDA label is enough to secure strong payer market access. This blog is the first installment in a series summarizing our findings. Where does it fit in treatment and how long is it used?

Marketing 52

Marketing FDA Approval FDA Production 52

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Could you give us an overview of the Lineage platform?

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Drug Target Review

SEPTEMBER 7, 2023

The first clinical candidate, BSB-1001, aims to transform leukemia treatment by targeting minor histocompatibility antigen-1. This two-part therapy aligns with allogeneic hematopoietic stem cell transplantation to enhance patient outcomes. TCX-102 complements this effort by focusing on TCRs against NPM1 gene mutations.

Treatment 98

Treatment Licensing Licensing Disease 98

Conversations in Drug Development Trends

JULY 9, 2024

The recent surge in GLP-1 treatments has ignited a profound shift in the pharmaceutical landscape, with more studies being announced than ever before. However, the treatment journey does not end with weight reduction, as maintaining weight loss overall is equally crucial.

Clinical Research 52

Clinical Research Treatment Research Clinical Trials 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

Vaccine 52

Vaccine Therapies Clinical Trials Small Molecule 52

The Pharma Data

MAY 15, 2023

CHMP Adopts Positive Opinion Recommending Hepcludex® (Bulevirtide) for Full Marketing Authorization for the Treatment of Hepatitis Delta Virus (HDV) Gilead Sciences, Inc. Bulevirtide was initially granted conditional marketing authorisation in July 2020 to provide people living with HDV urgent access to treatment.

Virus 40

Virus Marketing Treatment Science 40

Drug Target Review

AUGUST 6, 2024

Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.

Therapies 117

Therapies Pharmaceutical Companies Disease Treatment 117

PPD

JUNE 27, 2024

The PPD clinical research business of Thermo Fisher Scientific has made significant contributions to these indications — our experts have conducted pivotal clinical studies for at least four molecules now on the market. Accelerate your alopecia areata treatments.

Treatment 52

Treatment Clinical Trials Clinical Research Small Molecule 52

ASPET

SEPTEMBER 7, 2023

Alglucosidase alfa, a recombinant human acid α-glucosidase, was the first approved treatment for Pompe disease, but its uptake into skeletal muscle via the cation-independent mannose-6-phosphate (M6P) receptor (CIMPR) is limited. These results further substantiate the crucial role of CIMPR binding in lysosomal targeting of ERTs.

Small Molecule 100

Small Molecule Therapies Disease Treatment 100

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% Some of these treatments potentially make permanent changes to the humans they are introduced into.

Therapies 52

Therapies Research Clinical Trials Trials 52

The Pharma Data

MAY 27, 2022

In the pivotal clinical study CARTITUDE-1, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 80 percent of patients who responded experienced a stringent complete response . In December 2017, Janssen Biotech, Inc. to develop and commercialise cilta-cel.[1].

Therapies 52

Therapies Treatment Pharmaceutical Companies Nurses 52

Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies?

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

The Pharma Data

APRIL 27, 2022

NYSE: BHVN) today announced that the European Commission (EC) has granted marketing authorization for VYDURA® (rimegepant), a calcitonin gene-related peptide (CGRP) receptor antagonist for both the acute treatment of migraine with or without aura, and prophylaxis of episodic migraine in adults who have at least four migraine attacks per month.

Treatment 52

Treatment Marketing FDA Approval Production 52

ASPET

OCTOBER 12, 2023

The focus of the CET RAIDR effort is to bridge treatment gaps between threat identification and the implementation of licensed targeted MCMs, thereby strengthening warfighter resiliency. The CET RAIDR program conserves both time-to-market and funds by leveraging previous conventional development work as a launch point for repurposing efforts.

Therapies 100

Therapies Licensing Licensing FDA Approval 100

PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

Therapies 69

Therapies Immune Response DNA Virus 69

The Pharma Data

SEPTEMBER 10, 2021

– Abrocitinib is a once-daily oral JAK1 inhibitor indicated in Great Britain for the treatment of moderate to severe atopic dermatitis in patients aged 12 years and over, who are candidates for systemic therapy-. This is the first marketing authorization globally for abrocitinib-.

Marketing 52

Marketing Licensing Licensing Small Molecule 52

DS in Pharmatics

JUNE 10, 2023

On April 12, 2023, BeNeLuxA1 announced that negotiations failed to secure reimbursement for Libmeldy, a gene therapy developed by Orchard Therapeutics for the treatment of metachromatic leukodystrophy, on grounds of price. In the UK, an initial application to NICE was rejected as well back in 2020. This is a huge […]

Marketing 85

Marketing Therapies Treatment 85

Drug Target Review

OCTOBER 28, 2024

From R&D I moved into a marketing role which was very sales support oriented and included the roll-out of programmes, travelling across Europe and going into the field with sales reps who were almost all male at that time. At Terumo BCT we have been developing medical affairs and market access capabilities over the last several years.

Engineering 96

Engineering Therapies Treatment Disease 96

DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

Drug Development 71

Drug Development Metabolic Stability Clinical Trials Drugs 71

The Pharma Data

NOVEMBER 17, 2021

Biogen moment blazoned that the European Commission (EC) has granted marketing authorization for VUMERITY ® (diroximel fumarate) to treat grown-ups with relapsing- remitting multiple sclerosis (MS). In EVOLVE-MS-2, the rate of overall treatment termination was lower in actors treated with VUMERITY compared to those treated with TECFIDERA (1.6

Treatment 52

Treatment Marketing Clinical Trials Biosimilars 52

DrugBank

AUGUST 9, 2024

  The average cost of bringing a new drug to market is about $2.6 When a drug loses patent protection, generic or biosimilar versions can enter the market, leading to a rapid decline in sales for the original brand-name drug. Pharmaceutical Market Analysis The global pharmaceutical market revenue in 2024 is projected to be 1.15

Pharmaceuticals 77

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 77

Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

Research 59

Research Clinical Trials Therapies Immune Response 59

The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

Therapies 52

Therapies FDA Science Treatment 52

The Pharma Data

FEBRUARY 25, 2022

If approved, rimegepant will be the first oral CGRP receptor antagonist in the EU, and the only migraine medication approved for both acute and preventive treatment. Pfizer is proud to have a strong footprint in Europe, which will help bring this important potential new treatment option to millions of adults in Europe living with migraine.”.

Treatment 52

Treatment Small Molecule FDA Approval Therapies 52

The Pharma Data

FEBRUARY 24, 2022

Designation is supported by Phase II efficacy and safety data that will be presented at ATS 2022 Boehringer Ingelheim plans to study this novel investigational therapy in patients with progressive fibrosing interstitial lung diseases. The FDA Breakthrough Therapy designation is supported by data collected to date.

Therapies 52

Therapies FDA Clinical Trials Disease 52

The Pharma Data

APRIL 22, 2023

Bristol Myers Squibb Submits Application for Abecma®, a CAR T Cell Therapy, for Patients with Relapsed or Refractory Multiple Myeloma Who Have Received at Least Two Prior Therapies Bristol-Myers Squibb K.K. and Europe. Source link: [link]

Therapies 40

Therapies Treatment Virus Disease 40

LifeSciVC

OCTOBER 13, 2022

By Deanna Petersen, CBO of AVROBio, as part of the From the Trenches feature of LifeSciVC Gene therapy’s tremendous potential to transform the treatment of both rare and common diseases has been understood for decades. I believe we’re seeing this level of activity because gene therapy has reached a tipping point.

Therapies 40

Therapies Disease Treatment Marketing 40

The Pharma Data

AUGUST 19, 2021

. – If Authorized, Lenacapavir Would be the First Capsid Inhibitor and the Only HIV-1 Treatment Option Administered Twice Yearly –. Food & Drug Administration (FDA) approval for the treatment of HIV-1 infection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection in combination with other antiretrovirals.

Marketing 52

Marketing Treatment FDA FDA Approval 52

Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

Research 52

Research Clinical Trials Therapies Immune Response 52

The Pharma Data

SEPTEMBER 10, 2020

The EMA has asked for full data from an ongoing Phase III study of the treatment in patients with severe haemophilia A. European regulators have requested more data to review the marketing application for BioMarin’s haemophilia A gene therapy Valrox (valoctocogene roxaparvove).

Therapies 52

Therapies Regulations Treatment Marketing 52