Pharmacist Digest

STAT+: Day One drug for common childhood brain tumor approved by FDA

STAT

APRIL 23, 2024

D ay One Biopharmaceuticals said Tuesday the Food and Drug Administration approved its pill for one of the most common forms of childhood brain tumors, called pediatric low-grade glioma. The drug, previously known as tovorafenib, will be marketed as Ojemda. Day One has not yet disclosed a price.

Drug Development

SCOTUS’ abortion pill mifepristone case is really about the FDA

STAT

MARCH 25, 2024

WASHINGTON — The Supreme Court on Tuesday will hear opening arguments in an abortion medication case that pharmaceutical companies warn could upend the industry and paralyze new drug development. Now, in the wake of Roe v. Read the rest…

Drug Development

Drug Development Pharmaceutical Companies Pharmaceutical Companies

STAT+: I was wrong about Geron’s blood cancer drug. Now, is it a takeover target?

STAT

MARCH 18, 2024

For years, I’ve believed — and declared publicly — that Geron’s effort to develop a blood cancer drug called imetelstat would end in disappointment. The drug was never getting approved , I said. The strong endorsement makes an FDA approval highly likely.

The U.K. just cleared a drug to prevent breast cancer. U.S. women may not know it’s already an option

STAT

NOVEMBER 13, 2023

has approved a preventive drug for post-menopausal women at risk of developing breast cancer wasn’t a shock to breast medical oncologist Abenaa Brewster. The news that the U.K. But the fact that a patient in her breast cancer prevention clinic hadn’t ever heard of such a possibility before was.

STAT+: Life science leaders on how to make drug development less expensive, slow, and risky

STAT

MAY 7, 2024

Drug development is essentially a long, expensive bet: 90% of drugs fail during clinical trials, goes one of the life science industry’s most oft-quoted statistics. But they cautioned that the pace of future successes will depend on whether the industry can streamline and rethink drug development.

Drug Development

Drug Development Vaccines

A nonprofit group is pushing to get estrogen FDA approved for gender-affirming care

STAT

NOVEMBER 28, 2023

Hormone replacement therapies have long been approved by the Food and Drug Administration for use by cisgender patients, such as women who take estrogen when going through menopause. 17 in lieu of a live pre-investigational new drug meeting. 17 in lieu of a live pre-investigational new drug meeting.

Labelling

Opinion: STAT+: How payer-participating trials could help transform clinical research on FDA-approved drugs

STAT

OCTOBER 6, 2023

Earlier this year, Food and Drug Administration Commissioner Robert M. Califf proposed something radical: that private health care insurance companies (payers) should participate in clinical research on FDA-approved drugs. Bringing payers into clinical research could help solve these problems.

Drug Development

Listen: A dilemma in ALS, the first MASH drug, & why gene therapy is hard

STAT

MARCH 14, 2024

What happens when an approved drug doesn’t work? Bioethicist Holly Fernandez Lynch joins us to discuss the case of Amylyx Pharmaceuticals’ treatment for ALS and what its failure means for drug development. Why don’t patients want gene therapy? And is MASH still a big deal? Read the rest…

Drug Development

STAT+: Liver disease MASH is next target for obesity drugs in development

STAT

FEBRUARY 26, 2024

As drugmakers race to join the obesity drug market ignited by the approval of Wegovy and Zepbound , they’re not just competing on their drugs’ weight loss effects. They’re also competing on their products’ ability to treat a severe form of liver disease.

Diabetes

Diabetes Compounding Drug Development

STAT+: FDA expands approval of CRISPR-based medicine to treat beta thalassemia

STAT

JANUARY 16, 2024

The Food and Drug Administration on Tuesday approved a CRISPR-based medicine to treat beta thalassemia, an inherited blood disorder. The expanded approval of the therapy, called Casgevy, was widely expected but came two months ahead of the FDA’s decision deadline, known as a PDUFA date.

STAT+: As Humira biosimilars take over the market, CVS has created a new ploy: the drug ‘rebate credit’

STAT

MARCH 18, 2024

The biggest enticement that large pharmacy benefit managers offer to the employers that hire them is drug rebates — a steady stream of money sent back to their clients, a tangible symbol of the discounts that PBMs are able to wrangle out of pharmaceutical companies. Continue to STAT+ to read the full story…

STAT+: Madrigal wins first U.S. approval for drug to treat liver disease MASH

STAT

MARCH 14, 2024

The Food and Drug Administration on Thursday approved the first medicine developed specifically to treat the serious liver disease known as MASH. The pill, called Rezdiffra, is made by Madrigal Pharmaceuticals. Metabolic dysfunction-associated steatohepatitis, or MASH, is often associated with obesity.

Opinion: STAT+: It’s time to rethink everything about our approach to psychiatric drug development

STAT

MARCH 8, 2024

People experiencing mental health conditions are prescribed various drugs until one (or a combination) finally works — a painful process that can take years. Despite significant efforts, progress in psychiatric drug development has remained disappointingly slow. In many ways, psychiatry is still flying blind.

Drug Development

Q&A with Jennifer Adair, researcher on a mission to increase global access to gene therapies

STAT

MAY 9, 2024

These lags are ubiquitous in the current drug development system, where new therapies are approved in wealthy countries and then slowly leak out to low- and middle-income countries, many years later. BALTIMORE — Gene and cell therapies have transformed a handful of devastating disorders in the U.S.

Drug Development

Opinion: Aduhelm was a mess — and it could happen again

STAT

FEBRUARY 6, 2024

Last week, Biogen announced it will cease both the study and sale of Aduhelm, its FDA-approved monoclonal antibody for the treatment of Alzheimer’s disease. Its decision, the company explained, is not a response to new data about the drug’s safety or efficacy, but instead “a reprioritization of resources.”

STAT+: Novo Nordisk’s diabetes drug Ozempic cuts risk of kidney disease progression, trial shows

STAT

MARCH 5, 2024

LONDON — Novo Nordisk’s diabetes medication Ozempic cut the risk of patients developing advanced kidney disease or dying from kidney or heart complications, the company said Tuesday, adding to the evidence the wildly popular drug has broader health benefits for patients beyond addressing their diabetes. and Europe.

Diabetes

STAT+: Bristol Myers to purchase Karuna, maker of schizophrenia drug, for $14 billion

STAT

DECEMBER 22, 2023

Bristol Myers Squibb said Friday that it will acquire Karuna Therapeutics, a developer of medicines for neuropsychiatric conditions, for $14 billion. The centerpiece of the transaction is a drug called KarXT to treat schizophrenia. The deal values Karuna at $330 per share, or a 53% premium to its Thursday share price.

FDA calls for treatments for meth and cocaine addiction

STAT

OCTOBER 4, 2023

No medication is approved to treat addiction to either methamphetamine or cocaine. But the Food and Drug Administration is taking steps to fill that glaring gap in addiction medicine. While the country’s current drug overdose crisis is largely driven by opioids, stimulant use has skyrocketed in recent years.

STAT+: The inside story of Enhertu’s pan-tumor approval

STAT

APRIL 11, 2024

The inside story of Enhertu’s pan-tumor approval Cancer drug history was made last Friday. Enhertu, the blockbuster antibody-drug conjugate discovered by Daiichi Sankyo and co-developed by AstraZeneca, was approved in the U.S. Send me thoughts, comments, and questions to BiotechScorecard@statnews.com.

STAT+: First medicine developed to treat MASH expected to be approved soon

STAT

MARCH 12, 2024

The Food and Drug Administration is expected to approve the first medicine developed specifically to treat the serious liver disease known as MASH as soon as this week. The pill, called resmetirom, is made by Madrigal Pharmaceuticals.

Studies highlight risks of excluding people with obesity from drug trials

STAT

OCTOBER 16, 2023

People with obesity often go underrepresented in drug development trials, a critical gap that researchers say leaves drugmakers and doctors unsure of efficacy or risks in that patient population. “People with obesity are underrepresented in clinical trials for drug approval.” ” Read the rest…

Drug Development

STAT+: Bristol wins U.S. approval for lung cancer drug targeting rare genetic mutation

STAT

NOVEMBER 16, 2023

The Food and Drug Administration on Wednesday approved a new treatment for people with non-small cell lung cancer that contains a rare genetic alteration called Ros1. It was developed by a small biotech company, Turning Point Therapeutics, that was acquired by Bristol in June 2022.

STAT+: FDA agrees to review a rare disease drug that its developer was about to give up on

STAT

APRIL 8, 2024

Food and Drug Administration has agreed to review a rare disease drug developed by a small company that planned to shelve further work if the agency did not take this long-sought step. However, the FDA did not issue a priority review, which could delay approval. Continue to STAT+ to read the full story…

Drug Development

New Drug Approval for in-House Developed Anti-Insomnia Drug Dayvigo (Lemborexant) in Hong Kong

Pharma Mirror

MARCH 2, 2021

has obtained approval for the in-house-discovered and developed orexin receptor antagonist DAYVIGO (generic name: lemborexant) for the treatment of adults with insomnia, characterized by difficulties with sleep onset and/or sleep maintenance. This approval is the first approval for DAYVIGO in Asia outside of Japan.

STAT+: Bernie Sanders targets ‘outrageously high price’ of Ozempic

STAT

APRIL 25, 2024

The need-to-know this morning AstraZeneca reported first-quarter sales and profits that topped analyst estimates, buoyed by its blockbuster cancer drugs. Regeneron Pharmaceuticals and Mammoth Biosciences are partnering to develop new CRISPR-based gene therapies.

Hospitals

Hospitals Drug Pricing

STAT+: In reversal, FDA advisers vote to support approval of Amylyx’s drug for ALS

STAT

SEPTEMBER 8, 2022

At the end of an unusual and dramatic meeting on Wednesday, an independent panel of advisers to the Food and Drug Administration recommended the approval of a new drug to treat people with ALS developed by Amylyx Pharmaceuticals. Continue to STAT+ to read the full story…

STAT+: Gilead to acquire CymaBay for $4.3 billion, adding new treatment for liver disease

STAT

FEBRUARY 12, 2024

billion, adding a new treatment for a liver disease that is on track for approval later this year. That’s a relatively small takeout premium, but CymaBay shares have doubled in value since last fall on growing investor confidence in the company’s lead drug and speculation that the company could be acquired.

STAT+: Verve Therapeutics ‘pauses’ tests of its gene editor for heart disease and prioritizes a backup treatment

STAT

APRIL 2, 2024

Verve Therapeutics said Tuesday that it is pausing testing of its closely watched gene-editing treatment for high cholesterol after a patient receiving the drug experienced elevated liver enzymes and low platelet levels. Continue to STAT+ to read the full story…

Compounding

STAT+: Cognito raises $35 million for Alzheimer’s treatment device, touts benefits compared to drugs

STAT

JANUARY 29, 2024

Just as a new wave of approved drugs for Alzheimer’s disease reaches patients, medical device company Cognito Therapeutics has raised a fresh $35 million to advance its alternative treatment for the neurodegenerative disease. With the new Series B extension funding, the Cambridge, Mass.-based

Now a biopharma powerhouse, Vertex’s R&D bedrock could deliver tomorrow’s blockbusters

PharmaVoice

APRIL 30, 2024

From its landmark CRISPR approval to its potential breakthrough drug for pain, Vertex’s development approach, led by chief scientific officer Dr. David Altshuler, is paying off.

STAT+: FDA removes neurologist with financial ties to Eisai and Biogen from Alzheimer’s advisory panel

STAT

JUNE 5, 2023

The Food and Drug Administration has removed a neurologist with financial ties to Biogen and Eisai — which jointly develop and market a pair of controversial Alzheimer’s drugs — from an upcoming advisory panel meeting to review one of those treatments. Continue to STAT+ to read the full story…

STAT+: Pharmalittle: We’re reading about a CRISPR approval, selling meds directly to patients, and more

STAT

JANUARY 17, 2024

Food and Drug Administration approved a CRISPR-based medicine to treat beta thalassemia, an inherited blood disorder , STAT notes. The expanded approval of the therapy, called Casgevy, was widely expected but came ahead of a March 30 FDA deadline for a decision. for both approved indications. … The U.S.

STAT+: Years after a polarizing approval, Biogen walks away from Aduhelm

STAT

JANUARY 31, 2024

Biogen is giving up its ownership of Aduhelm , the Alzheimer’s disease treatment whose 2021 approval led to scrutiny and outrage, turning the page on a tempestuous chapter in the company’s long history. ” Continue to STAT+ to read the full story…

FDA approves Alzheimer’s drug shown to moderately slow cognitive decline in early stages of the disease

STAT

JANUARY 6, 2023

The Food and Drug Administration on Friday approved a new Alzheimer’s disease treatment that moderately slows cognitive decline in people with early-stage disease. Read the rest…

Pharmaceutical Companies

Pharmaceutical Companies Pharmaceutical Companies Drug Pricing

Opinion: STAT+: A California court is setting a dangerous precedent over drug development (or lack thereof) liability

STAT

FEBRUARY 13, 2024

The assault on the development of new lifesaving therapies continues apace. Recently, the First Appellate Division of California Appeals held that companies not only have to defend products they have developed and marketed, but also those they have not. Ultimately, this medicine was shown to have a better side effect profile than TDF.

Drug Development

Opinion: STAT+: Use evidence to support early coverage of gene therapy after accelerated approval

STAT

APRIL 23, 2024

Gene editing and therapy to replace missing or defective genes is one of the most exciting recent medical developments, offering tremendous hope for people with rare diseases and genetic conditions. The Food and Drug Administration has approved several gene therapies for rare diseases through the accelerated pathway.

STAT+: FDA has some safety concerns about CRISPR-based sickle cell treatment, documents show

STAT

OCTOBER 27, 2023

The Food and Drug Administration said on Friday that it has some safety concerns about an experimental CRISPR-based treatment for sickle cell disease, citing the methods used by its makers to evaluate the risk of inadvertently making unwanted changes to patients’ DNA. Continue to STAT+ to read the full story…

Documentation

STAT+: European antitrust regulators probe Zoetis over pain drug for dogs

STAT

MARCH 26, 2024

Librela was approved by European regulators in 2020 and in the U.S., But at the same time that Zoetis was developing its drug, it acquired another late-stage treatment for the same use, and this was supposed to be sold in the European Economic Area by another company. where Zoetis is headquartered, in 2023.

STAT+: FDA approves a gene therapy for the inherited bleeding disorder hemophilia B

STAT

NOVEMBER 22, 2022

The Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder. The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company.

Pharmaceutical Companies

Pharmaceutical Companies Pharmaceutical Companies

STAT+: FDA approves first Alzheimer’s therapy shown to clearly slow cognitive decline

STAT

JULY 6, 2023

The Food and Drug Administration on Thursday granted full approval to the first therapy for Alzheimer’s disease clearly shown to slow the cognitive decline associated with the disease — a milestone in treatment, even if the benefits are modest. It previously secured conditional approval in January.

Pharmaceutical Companies

Pharmaceutical Companies Pharmaceutical Companies

STAT+: FDA advisory panel recommends conditional approval of Biogen’s drug for rare form of ALS

STAT

MARCH 22, 2023

An independent panel of advisors to the Food and Drug Administration on Wednesday concluded that a treatment developed by Biogen for a rare, genetic form of ALS should be approved, despite unanswered questions about its benefit to patients. Continue to STAT+ to read the full story…

STAT+: Pharmalittle: We’re reading about Biocon eyeing weight loss drugs, Sanofi layoffs, and more

STAT

APRIL 8, 2024

Food and Drug Administration agreed to review an application for a rare disease drug developed by a small company that planned to shelve further work if the agency did not take this long-sought step , STAT says. The FDA will also hold an advisory committee meeting.

Drug Development

STAT+: Evelo Biosciences, a microbiome company launched by Flagship, shuts down

STAT

NOVEMBER 22, 2023

Evelo Biosciences, a startup hoping to use the trillions of microbes in and on the human body to fight disease, is shutting down after running low on cash and seeing multiple drug trial failures. The field has developed a couple of drugs. diff was approved in April. The biotech, which was based in Cambridge, Mass.,

FDA approves first RSV vaccine, a long-sought scientific achievement

STAT

MAY 3, 2023

The Food and Drug Administration on Wednesday licensed the first-ever vaccine for respiratory syncytial virus, or RSV , completing an elusive quest that has been decades in the making. The product, GSK’s Arexvy, was approved for adults ages 60 and older. GSK beat a crowded field of competitors to cross the finish line first.

Vaccines

STAT+: Day One drug for common childhood brain tumor approved by FDA

SCOTUS’ abortion pill mifepristone case is really about the FDA

Trending Sources

STAT+: I was wrong about Geron’s blood cancer drug. Now, is it a takeover target?

The U.K. just cleared a drug to prevent breast cancer. U.S. women may not know it’s already an option

STAT+: Life science leaders on how to make drug development less expensive, slow, and risky

A nonprofit group is pushing to get estrogen FDA approved for gender-affirming care

Opinion: STAT+: How payer-participating trials could help transform clinical research on FDA-approved drugs

Listen: A dilemma in ALS, the first MASH drug, & why gene therapy is hard

STAT+: Liver disease MASH is next target for obesity drugs in development

STAT+: FDA expands approval of CRISPR-based medicine to treat beta thalassemia

STAT+: As Humira biosimilars take over the market, CVS has created a new ploy: the drug ‘rebate credit’

STAT+: Madrigal wins first U.S. approval for drug to treat liver disease MASH

Opinion: STAT+: It’s time to rethink everything about our approach to psychiatric drug development

Q&A with Jennifer Adair, researcher on a mission to increase global access to gene therapies

Opinion: Aduhelm was a mess — and it could happen again

STAT+: Novo Nordisk’s diabetes drug Ozempic cuts risk of kidney disease progression, trial shows

STAT+: Bristol Myers to purchase Karuna, maker of schizophrenia drug, for $14 billion

FDA calls for treatments for meth and cocaine addiction

STAT+: The inside story of Enhertu’s pan-tumor approval

STAT+: First medicine developed to treat MASH expected to be approved soon

Studies highlight risks of excluding people with obesity from drug trials

STAT+: Bristol wins U.S. approval for lung cancer drug targeting rare genetic mutation

STAT+: FDA agrees to review a rare disease drug that its developer was about to give up on

New Drug Approval for in-House Developed Anti-Insomnia Drug Dayvigo (Lemborexant) in Hong Kong

STAT+: Bernie Sanders targets ‘outrageously high price’ of Ozempic

STAT+: In reversal, FDA advisers vote to support approval of Amylyx’s drug for ALS

STAT+: Gilead to acquire CymaBay for $4.3 billion, adding new treatment for liver disease

STAT+: Verve Therapeutics ‘pauses’ tests of its gene editor for heart disease and prioritizes a backup treatment

STAT+: Cognito raises $35 million for Alzheimer’s treatment device, touts benefits compared to drugs

Now a biopharma powerhouse, Vertex’s R&D bedrock could deliver tomorrow’s blockbusters

STAT+: FDA removes neurologist with financial ties to Eisai and Biogen from Alzheimer’s advisory panel

STAT+: Pharmalittle: We’re reading about a CRISPR approval, selling meds directly to patients, and more

STAT+: Years after a polarizing approval, Biogen walks away from Aduhelm

FDA approves Alzheimer’s drug shown to moderately slow cognitive decline in early stages of the disease

Opinion: STAT+: A California court is setting a dangerous precedent over drug development (or lack thereof) liability

Opinion: STAT+: Use evidence to support early coverage of gene therapy after accelerated approval

STAT+: FDA has some safety concerns about CRISPR-based sickle cell treatment, documents show

STAT+: European antitrust regulators probe Zoetis over pain drug for dogs

STAT+: FDA approves a gene therapy for the inherited bleeding disorder hemophilia B

STAT+: FDA approves first Alzheimer’s therapy shown to clearly slow cognitive decline

STAT+: FDA advisory panel recommends conditional approval of Biogen’s drug for rare form of ALS

STAT+: Pharmalittle: We’re reading about Biocon eyeing weight loss drugs, Sanofi layoffs, and more

STAT+: Evelo Biosciences, a microbiome company launched by Flagship, shuts down

FDA approves first RSV vaccine, a long-sought scientific achievement

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