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.

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FDA 96

Fierce Pharma

NOVEMBER 7, 2023

Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Vertex and CRISPR Therapeutics are up first with a Dec. |

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MedCity News

APRIL 26, 2024

The FDA has approved Beqvez, a Pfizer gene therapy developed for moderate-to-severe hemophilia B. million price tag, the same as a CSL Behring gene therapy already available for treating the inherited bleeding disorder. The one-time treatment carries a $3.5

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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.

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FDA 105

Pharmaceutical Technology

NOVEMBER 9, 2022

On November 7, the company announced that the FDA has scheduled a pre-licensure inspection of BioMarin’s manufacturing facility, and required the company to submit three-year Phase III Roctavian data in the approval application. Since its FDA approval in August 2022, the beta-thalassemia therapy was launched in the US with a $2.8

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pharmaphorum

OCTOBER 3, 2022

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

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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.

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FDA 52

MedCity News

JULY 1, 2022

Novartis is acquiring an FDA priority review voucher from Mallinckrodt Pharmaceuticals. The $100 million price tag is in the neighborhood of the going rate for these vouchers, which grant a company a shorter regulatory review timeline for a drug that addresses a rare or neglected disease.

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World of DTC Marketing

JUNE 23, 2021

One agency FDA adviser, resigning from his committee post in protest, called it “probably the worst drug approval decision in recent U.S. Biogen’s Aduhelm won FDA approval after discovering that some patients on higher dosages had displayed improvement of.39 Not to mention the $56,000 annual price tag.

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Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Shortly after this, the FDA announced the accelerated approval of bluebird's Skysona (elivaldogene autotemcel), or eli-cel, on 19 September. They usually face resistance from insurers and may struggle to gain coverage at launch.

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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.

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FDA 52

Pharmaceutical Technology

OCTOBER 9, 2023

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

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FDA 52

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.

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FDA 96

Pharmaceutical Technology

JUNE 23, 2023

Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.

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FDA 52

pharmaphorum

AUGUST 21, 2022

Shares in Axsome Therapeutics have rocketed on FDA approval of its depression therapy Auvelity (formerly AXS-05) – a year after its approval was held up by the regulator. The post Axsome bounces back as FDA clears depression drug appeared first on. Following after is AXS-12 (reboxetine) for narcolepsy, which could be filed in 2023.

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pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

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World of DTC Marketing

SEPTEMBER 24, 2021

Then, against the advisory panel’s advice, Aduhelm was approved, but insiders within the industry criticized both the FDA and Biogen weren’t sold on the drug or its price tag. After an unstable period, they lost their CEO and some very good people who decided to abandon ship.

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World of DTC Marketing

SEPTEMBER 7, 2022

In pharma, growth depends on new products with hefty price tags when over 80% of voters want lower costs for their prescription drugs. Wall Street wants growth, as do investors, but that may be impossible for pharmaceutical companies. Biogen’s failure should be a warning to other pharma companies. ” Why are the costs so high?

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pharmaphorum

AUGUST 25, 2022

Talquetamab has also shown promise in myeloma in the phase 1 MonumenTAL-1 study, which showed a 70% overall response rate (ORR) with the drug, and claimed a breakthrough designation from the FDA in June. A marketing application for teclistamab is also under review at the FDA, with a decision expected later this month or in early September.

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FDA Marketing Safety Patients 93

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.

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pharmaphorum

DECEMBER 12, 2022

Vyvgart is already approved to treat generalised myasthenia gravis, getting a green light for that indication from the FDA a year ago, and launched with a $225,000 per annum price tag in competition with rival gMG drugs like AstraZeneca/Alexion’s C5 inhibitor Soliris (eculizumab).

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pharmaphorum

JULY 25, 2022

Tecvayli (teclistamab) is an off-the-shelf bispecific antibody targeting both BCMA and CD3 which is also under review at the FDA and if approved will slot into J&J’s myeloma therapy portfolio alongside Darzalex (daratumumab) – an antibody directed at CD38 – and BCMA-targeted CAR-T therapy Carvykti (ciltacabtagene autoleucel). .”

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Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.

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World of DTC Marketing

DECEMBER 13, 2021

As cancer survival rates rise, so do the price tags of life-saving treatments. Few cancer drugs approved via the accelerated FDA approval pathway were judged to have verified benefits based on improvement in survival reported in confirmatory trials. OPENING: American cancer patients spent more than $21 billion on their care in 2019.

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Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing. Since the price of the therapies is unknown, the institute used a placeholder cost of $2 million.

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Pharmaceutical Technology

FEBRUARY 22, 2023

Developed by Sanofi and Regeneron, Praluent was approved by the FDA in July 2015. Both drugs come with a high price tag. Amgen’s Repatha was greenlit a month later. Both drugs are monoclonal antibodies that inhibit the protein PCSK9.

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Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. It will be six years since the US FDA approval of Kymriah in August this year.

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Pharmaceutical Technology

MARCH 22, 2023

The pharmaceutical industry began using radio frequency identification (RFID) tags in the early 2000s. Pfizer was the first to use the tech, adding RFID tags to track a Viagra (sildenafil) shipment circa 2006. Twenty years ago, the cost of implementing RFID tags and the ecosystem (software) was much more expensive than today.

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Food and Drug Administration Electronics Pharma FDA 105

Contrarian Sales Techniques

JANUARY 23, 2024

The map leads you through regulatory landscapes, navigating intricate FDA and compliance guidelines. Some found the material dense, the exam daunting, and the price tag a tad hefty. It delves into the science behind the drugs, equipping you with knowledge of anatomy, physiology, and therapeutic areas. But here's the thing.

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PM360

OCTOBER 28, 2022

Automation helps here, establishing patterns and design systems and standards that can be pre-approved and FDA compliant. In addition, content platforms enable automated and streamlined management of asset metadata, tagging, usage tracking, and searchability.

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Pharmaceutical Technology

JUNE 21, 2023

The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. Despite the limited market, the price tags of haemophilia make it potentially lucrative. Haemophilia B is a rare condition.

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Patients Food and Drug Administration Engineering Sales 98

pharmaphorum

AUGUST 26, 2022

It’s a major step forward for BioMarin after its first attempt to secure approval for Roctavian in the US was knocked back, with the FDA rejecting its application in 2020 with a request for more data on the durability of its effects. BioMarin has said Roctavian will be available at a ‘net’ price of around €1.5

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FDA Marketing Competition Patients 110

European Pharmaceutical Review

MAY 11, 2023

Three SMN-enhancing treatments for the condition are approved by the US Food and Drug Administration (FDA) 2 , however there is no cure for SMA at present. While Zolgensma is also being investigated in the form of an injection, the MOA currently approved by the FDA is a single infusion. percent of Europe’s annual healthcare spend.

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Pharmaceutical Technology

JANUARY 27, 2023

In 2019, Cablivi became the first FDA-approved , nanobody-based medicine for acquired thrombotic thrombocytopenic purpura (aTTP), an ultra-orphan condition that causes blood clots in small blood vessels around the body. In 2022, the number of risk sharing agreements peaked with 144 agreements being made in the pharmaceutical industry.

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PM360

SEPTEMBER 7, 2023

Mason learned that lesson while working with argenx to launch VYVGART (efgartigimod alfa-fcab), the first FDA-approved treatment of its kind for adults with anti-AChR antibody positive generalized myasthenia gravis (gMG), a rare autoimmune neuromuscular disease. We also put big pull tags on the little pouches that held the items.

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European Pharmaceutical Review

DECEMBER 27, 2023

This is in addition to its considerable price tag. FDA; 2022 [cited 2023 Mar]. The demand for CAR T-cell therapy is therefore much greater than the supply, making it difficult for patients to access the potentially life-saving treatment. Overview of approved car‐t therapies, ongoing clinical trials, and its impact on clinical practice.

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Cadensee

JANUARY 1, 2022

It's already tagged by clinicians with the diagnosis, so you have to do the machine learning and the engineering side. We're working with FDA hand-in-hand to make that highly accurate and highly safe. Starting with the FDA and not the C side. Whether it's a CT scans, X-rays, and so forth. TytoCare also creates the data.

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