Drugs - Clinical Research Informer

Lutris wins FDA orphan drug tag for EGFR inhibitor-induced rash candidate

Pharmaceutical Technology

FEBRUARY 29, 2024

LUT014 is being investigated in an ongoing Phase II trial as a first-line treatment for EGFR inhibitor-induced acneiform rash, for which there is no approved drug.

Drugs

Drugs Trials

Biogen’s Alzheimer’s drug: What’s the price tag on hope?

World of DTC Marketing

JUNE 1, 2021

SUMMARY: The FDA is coming under intense pressure to approve Biogen’s Alzheimer’s drug, but Aaron S. ” For Biogen, the stakes are high as approval is likely to earn tens of millions, of to billions, in profits but is hope a reason to approve a drug over science? .” Biogen needs a new drug revenue badly.

Drugs

Drugs Trials Doctors Doctor

Disc wins orphan drug tag for rare blood cancer

Pharmaceutical Technology

FEBRUARY 12, 2024

The humanised monoclonal antibody DISC-3405 is under investigation in a Phase I clinical trial, with data expected this year.

Clinical Trials

Clinical Trials Clinical Trials Antibody Drugs

Cybin bags breakthrough tag for psychedelic depression drug

pharmaphorum

MARCH 14, 2024

Cybin files a $150m placement on the back of new clinical data with its psychedelic drug CYB003 for depression and an FDA breakthrough designation.

Drugs

Revolo advances plans to tackle eosinophilic esophagitis with orphan drug tag

Pharmaceutical Technology

JANUARY 31, 2024

The FDA has granted an orphan drug designation to Revolo’s ‘1104, with plans for a Phase IIb study later this year.

Drugs

Sanders takes aim at US drug prices of Novo’s Ozempic and Wegovy

Pharmaceutical Technology

APRIL 25, 2024

A Senate Committee is investigating the “outrageously high” US price tags of the blockbuster diabetes and obesity drugs.

Drugs

Ultimovacs bags orphan drug tag for mesothelioma vaccine

Pharmaceutical Technology

OCTOBER 9, 2023

The FDA decision follows a similar designation for the vaccine UV1 against malignant melanoma in December 2021.

Vaccination

Vaccination Vaccine Drugs

Grünenthal’s non-opioid pain drug gets breakthrough tag

pharmaphorum

MAY 23, 2023

Grünenthal’s non-opioid pain drug gets breakthrough tag Phil.Taylor Tue, 05/23/2023 - 08:20 Bookmark this

Drugs

Harmony wins orphan drug tag for pitolisant to treat Prader-Willi syndrome

Pharmaceutical Technology

FEBRUARY 22, 2024

Harmony is advancing pitolisant into a Phase III clinical trial that is expected to start in Q1 2024.

Clinical Trials

Clinical Trials Clinical Trials Drugs Trials

NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug

Pharmaceutical Technology

OCTOBER 17, 2023

The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.

Drugs

Drugs Trials

NeoImmuneTech bags orphan drug tag for IL-7 acute radiation syndrome treatment

Pharmaceutical Technology

NOVEMBER 20, 2023

NeoImmuneTech is developing NT-17, an IL-7 treatment, to promote rapid T-cell recovery in patients with acute radiation syndrome.

Drugs

Drugs Development

Click bags FDA breakthrough tag for schizophrenia DTx

pharmaphorum

JANUARY 4, 2024

Digital health company Click Therapeutics has won an FDA breakthrough device designation for CT-155, a digital therapeutic (DTx) for the negative symptoms of schizophrenia, like social withdrawal and apathy, that tend not to respond well to antipsychotic drugs.

Drugs

Sarepta wins FDA approval of another drug for rare muscular dystrophy

Bio Pharma Dive

FEBRUARY 25, 2021

Like other Sarepta drugs, it comes with a high price tag and its clinical benefits haven't been confirmed. Amondys 45 is now cleared for the estimated 8% of Duchenne patients eligible for treatment.

FDA Approval

FDA Approval Drugs

Genprex’s Reqorsa gene therapy picks up orphan drug designation from FDA for SCLC

Pharmaceutical Technology

AUGUST 11, 2023

The latest tag adds to three fast track designations for Reqorsa, with the company initiating a Phase I/II trial in Q4 2023.

Gene Therapy

Gene Therapy Gene Trials Drugs

Amylyx ALS drug draws criticism over $158,000 price tag

pharmaphorum

OCTOBER 3, 2022

The third ALS treatment to be given FDA approval, following Mitsubishi Tanabe’s Radicava ($170,000 per year) and generic drug riluzole – Relyvrio is priced at around $12,500 per 28-day prescription. Currently, the ALS drug is priced about five times higher than the top of a value-based range. ALS affects over 30,000 people in the US.

Drugs

Drugs Generic Drugs FDA Approval Trials

‘Click’ chemistry used to tag proteins made by cancer cells

Drug Discovery World

OCTOBER 26, 2022

The new method involves adding chemical tags to sugar molecules which are added to cells. When the cells make these proteins, they remain marked with the chemical tag, meaning that researchers can identify them. . The post ‘Click’ chemistry used to tag proteins made by cancer cells appeared first on Drug Discovery World (DDW).

Protein

Protein Genetics Containment Research

Pfizer and Arvinas win UK’s fast track tag for breast cancer drug

Pharmaceutical Technology

AUGUST 1, 2023

Vepdegestrant was awarded the Innovation Passport designation for treating advanced or metastatic breast cancer.

Drugs

FDA gives Daiichi Sankyo’s HER3 drug a breakthrough tag

pharmaphorum

DECEMBER 23, 2021

Daiichi Sankyo has been granted breakthrough status by the FDA for patritumab deruxtecan, a HER3-targeted antibody-drug conjugate (ADC) in clinical trials for lung cancer. HER3 is expressed in more than 80% of EGFR-mutant NSCLC, and overexpression is associated with poor outcomes, but no HER3-targeted drugs have been approved to date.

Drugs

Drugs Antibody Clinical Trials Clinical Trials

BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds

Bio Pharma Dive

AUGUST 28, 2020

A draft report from the drug pricing watchdog concluded that, under the right conditions, Roctavian may be cost-effective at a price of $2.5

Gene Therapy

Gene Therapy Gene Drugs

Japan's Ono pays $2.4B for Qinlock-maker Deciphera as biotech readies another approval run

Fierce Pharma

APRIL 29, 2024

The price tag includes both the approved cancer drug Qinlock and another Deciphera candidate heading to the FDA’s desk in the coming months.

Drugs

Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US

Fierce Pharma

MARCH 20, 2024

After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S.,

Gene Therapy

Gene Therapy Gene Genetic Disease Genetics

FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag

pharmaphorum

JANUARY 5, 2022

The FDA thinks an antibody-drug conjugate (ADC) developed by AbbVie could set new standards in treatment for certain patients with lung cancer, awarding the drug breakthrough status. Hutchmed’s drug is also being paired with Tagrisso and AZ’s PD-L1 blocker Imfinzi (durvalumab) in global registrational trials.

Antibody

Antibody Drugs Protein Gene

After filing hitch, Eisai and Biogen begin rolling FDA submission for subcutaneous Leqembi

Fierce Pharma

MAY 14, 2024

regulators last month, Eisai and Biogen’s effort to deploy a more convenient version of their Alzheimer’s disease drug Leqembi appears to be back on track. After suffering a setback with U.S.

Regulation

Regulation Drugs

#news #biotech Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug

BioTech 365

JULY 20, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug.Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug … Continue reading →

Drugs

Drugs Research

Enhertu gets breakthrough tag in HER2-low breast cancer

pharmaphorum

APRIL 27, 2022

AstraZeneca and Daiichi Sankyo have claimed a fifth breakthrough designation from the FDA for Enhertu, shortly after showing the drug extended survival in patients with HER2-low metastatic breast cancer. Drugs given the designation are hastened through the development process by the FDA, which can grant them a faster six-month review.

Sales

Sales Drugs Antibody Licensing

DRUG REPURPOSING : A REVIEW

Pharma Tutor

OCTOBER 13, 2022

DRUG REPURPOSING : A REVIEW. Read more about DRUG REPURPOSING : A REVIEW Log in or register to post comments Read more about DRUG REPURPOSING : A REVIEW Log in or register to post comments Email : charmiagarwal823@gmail.com. Thu, 10/13/2022 - 15:56. Review Articles.

Pharmacy

Pharmacy Drugs

Blue Note leukaemia DTx gets FDA breakthrough tag

pharmaphorum

JANUARY 11, 2022

It is well-established for conventional drugs and medical devices but has only recently started to be used for DTx. The post Blue Note leukaemia DTx gets FDA breakthrough tag appeared first on. Breakthrough designations can speed up the development of new therapies, making them available to patients sooner.

Development

Development Production Drugs

FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag

pharmaphorum

JUNE 8, 2021

The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms” in approving Biogen’s Alzheimer’s disease drug Aduhelm. It said the drug was the first to treat the underlying mechanism in Alzheimer’s rather than addressing symptoms.

Drugs

Drugs Marketing FDA Approval Trials

STAT+: The FDA has approved a new Alzheimer’s drug, but wide access may depend on CMS easing restrictions

STAT News

JANUARY 6, 2023

Food and Drug Administration has granted accelerated approval to the widely anticipated Alzheimer’s drug from Eisai and Biogen, a key question is the extent to which payers — private and public — will cover the treatment. Such decisions are based on myriad factors, starting with the average $26,500 price tag.

Drugs

Drugs Clinical Trials Clinical Trials Medicine

STAT+: Medicare will take R&D costs, effectiveness into account when it negotiates drug prices. But studies show that doesn’t affect prices

STAT News

MARCH 10, 2023

By law, Medicare will have to take a medication’s efficacy and its research and development costs into account when it starts to negotiate drug prices — but recent research shows pharma companies ignore those factors when they pick prices for their products. But neither factor has much effect, the studies concluded.

Drugs

Drugs Pharma Companies Research Production

Roche nabs breakthrough tag for TIGIT cancer immunotherapy

pharmaphorum

JANUARY 5, 2021

Roche’s closely-watched combination of two checkpoint inhibitors – TIGIT-targeting tiragolumab and PD-L1 drug Tecentriq – has claimed breakthrough status from the FDA. Roche’s drug is already FDA-approved as a monotherapy and in combination with Avastin (bevacizumab) and chemotherapy for previously-untreated NSCLC with high PD-L1 expression.

Immune Response

Immune Response FDA Approval Drugs Sales

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. bluebird then broke its own pricing record when the $3m Skysona was approved, and now faces the unique challenge of selling the world's two most expensive drugs.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

Gilead tags Merck for first-line triple negative breast cancer trial

pharmaphorum

OCTOBER 29, 2021

Gilead’s Trop-2 targeting antibody-drug conjugate Trodelvy (sacituzumab govitecan) will be paired with Merck’s PD-1 inhibitor Keytruda (pembrolizumab) in a new phase 3 trial in previously-untreated subjects with locally advanced or metastatic TNBC. While its headline revenue of $7.4

Trials

Trials Sales FDA Approval Production

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Drugs

Drugs FDA Approval Marketing Development

Narcan's over-the-counter price will still put it out of reach for many, experts say

Medical Xpress

APRIL 21, 2023

The life-saving drug "Narcan" is expected to be available over-the-counter later this year, but its price tag is expected to put it out of reach for many below the poverty line.

Drugs

A REVIEW ON COMPUTER AIDED DRUG DESIGN (CAAD) AND IT’S IMPLICATIONS IN DRUG DISCOVERY AND DEVELOPMENT PROCESS

Pharma Tutor

JULY 13, 2022

A REVIEW ON COMPUTER AIDED DRUG DESIGN (CAAD) AND IT’S IMPLICATIONS IN DRUG DISCOVERY AND DEVELOPMENT PROCESS. Read more about A REVIEW ON COMPUTER AIDED DRUG DESIGN (CAAD) AND IT’S IMPLICATIONS IN DRUG DISCOVERY AND DEVELOPMENT PROCESS Log in or register to post comments About Authors. Sayyed , Ms. Review Articles.

Development

Development Drugs Pharmacy

Drug Regulatory Affairs Certificate Institutes in India

Pharma Tutor

JULY 18, 2022

Drug Regulatory Affairs Certificate Institutes in India. Historical Overview of Pharmaceutical Industries and Regulatory Affairs:During 1950s, multiple tragedies i.e., sulfanilamide elixir, vaccine tragedy and thalidomide tragedy have resulted in substantial increase of legislations for drug products quality, safety and efficacy.

Drugs

Drugs Manufacturing Vaccination Vaccine

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

pharmaphorum

DECEMBER 24, 2021

The anti-amyloid drug, which also claimed a breakthrough tag from the FDA in July, is being developed for the treatment of early-stage Alzheimer’s disease. The $56,000-a-year price tag for the drug, which has now been cut in half in the US, also hasn’t helped the drugmakers make a case for Aduhelm with US payers.

Drugs

Drugs FDA Approval Trials Marketing

Drug-delivery microcapsules tagged with zirconium-89 can be tracked by PET imaging

Scienmag

JANUARY 21, 2021

If these hollow capsules are modified to target a solid tumor, PET imaging and therapeutic ultrasound can be used to rupture them and release an anticancer drug at ground zero. Credit: UAB BIRMINGHAM, Ala.

Drug Delivery

Drug Delivery Drugs Medicine

Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

JULY 14, 2022

The commercial investment required to research and develop an innovative drug, prove its safety and efficacy, and bring it to market is staggering. Most famously, the US passed its Orphan Drug Act in 1983, providing innovators with financial motivation to develop orphan drugs and meet the needs of these forgotten patients.

Drugs

Drugs Medicine Marketing Development

New approach to improving clinical trial enrollment and diversity

Medical Xpress

MARCH 8, 2023

Failure to enroll enough participants in trials can delay the arrival of new therapies in the clinic and inflate their eventual price tags. For example, a drug that proves effective for White patients might not work as well in people of other races or ethnicities.

Clinical Trials

Clinical Trials Clinical Trials Trials Drugs

In Tough Market, Inventiva's NASH Drug Receives Breakthrough Therapy Tag

BioSpace

OCTOBER 12, 2020

NASH is a metabolic disease similar to cirrhosis of the liver, but occurs in people who drink little, if any, alcoholic.

Marketing

Marketing Drugs

Drug-resistant epilepsy drug Ontozry backed by NICE

pharmaphorum

NOVEMBER 16, 2021

NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma’s Ontozry. An opinion from the Scottish Medicine Consortium (SMC) on the use of the drug by NHS Scotland is expected to be published early in 2022.

Drugs

Drugs Medicine Clinical Trials Clinical Trials

STAT+: Drugmaker raises the price of an old chemo medicine tenfold amid persistent shortages

STAT News

OCTOBER 27, 2022

Amid sporadic shortages of a drug that is essential in preparing patients for lifesaving, cancer-fighting treatments, one manufacturer has returned to the market — but is selling its medicine for 10 to 20 times the prices offered by the only other companies with available supplies. Continue to STAT+ to read the full story…

Medicine

Medicine Manufacturing Marketing Drugs

Lutris wins FDA orphan drug tag for EGFR inhibitor-induced rash candidate

Biogen’s Alzheimer’s drug: What’s the price tag on hope?

Trending Sources

Disc wins orphan drug tag for rare blood cancer

Cybin bags breakthrough tag for psychedelic depression drug

Revolo advances plans to tackle eosinophilic esophagitis with orphan drug tag

Sanders takes aim at US drug prices of Novo’s Ozempic and Wegovy

Ultimovacs bags orphan drug tag for mesothelioma vaccine

Grünenthal’s non-opioid pain drug gets breakthrough tag

Harmony wins orphan drug tag for pitolisant to treat Prader-Willi syndrome

NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug

NeoImmuneTech bags orphan drug tag for IL-7 acute radiation syndrome treatment

Click bags FDA breakthrough tag for schizophrenia DTx

Sarepta wins FDA approval of another drug for rare muscular dystrophy

Genprex’s Reqorsa gene therapy picks up orphan drug designation from FDA for SCLC

Amylyx ALS drug draws criticism over $158,000 price tag

‘Click’ chemistry used to tag proteins made by cancer cells

Pfizer and Arvinas win UK’s fast track tag for breast cancer drug

FDA gives Daiichi Sankyo’s HER3 drug a breakthrough tag

BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds

Japan's Ono pays $2.4B for Qinlock-maker Deciphera as biotech readies another approval run

Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US

FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag

After filing hitch, Eisai and Biogen begin rolling FDA submission for subcutaneous Leqembi

#news #biotech Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug

Enhertu gets breakthrough tag in HER2-low breast cancer

DRUG REPURPOSING : A REVIEW

Blue Note leukaemia DTx gets FDA breakthrough tag

FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag

STAT+: The FDA has approved a new Alzheimer’s drug, but wide access may depend on CMS easing restrictions

STAT+: Medicare will take R&D costs, effectiveness into account when it negotiates drug prices. But studies show that doesn’t affect prices

Roche nabs breakthrough tag for TIGIT cancer immunotherapy

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Gilead tags Merck for first-line triple negative breast cancer trial

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Narcan's over-the-counter price will still put it out of reach for many, experts say

A REVIEW ON COMPUTER AIDED DRUG DESIGN (CAAD) AND IT’S IMPLICATIONS IN DRUG DISCOVERY AND DEVELOPMENT PROCESS

Drug Regulatory Affairs Certificate Institutes in India

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

Drug-delivery microcapsules tagged with zirconium-89 can be tracked by PET imaging

Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

New approach to improving clinical trial enrollment and diversity

In Tough Market, Inventiva's NASH Drug Receives Breakthrough Therapy Tag

Drug-resistant epilepsy drug Ontozry backed by NICE

STAT+: Drugmaker raises the price of an old chemo medicine tenfold amid persistent shortages

Stay Connected