PharmaShots

MAY 26, 2023

Takeda and HUTCHMED's Fruquintinib Receives Priority Review from the US FDA to Treat Metastatic Colorectal Cancer Date: May 26, 2023 | Tags: Takeda, HUTCHMED, Fruquintinib, Metastatic Colorectal Cancer, Regulatory, Priority Review, US, FDA Gilead Receives EMA’s CHMP Positive Opinion to Extend the Use of Veklury (remdesivir) for COVID-19 Date: (..)

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FDA 40

Pharmaceutical Technology

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Prior to the program, only 10 drugs were approved for a rare disease.

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PharmaShots

FEBRUARY 24, 2023

Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive Positive Solid Tumors Date: Feb 23, 2023 | Tags: Keymed, Lepu Biopharma, AstraZeneca, CMG901, Claudin 18.2-Positive

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PharmaShots

MAY 12, 2023

Sanofi Reports P-IIIb Trial (HARMONIE) Results of Nirsevimab for the Prevention of Hospitalizations due to RSV-Related LRTD Date: May 12, 2023 | Tags: Sanofi, Nirsevimab, RSV-Related LRTD, Clinical Trial, P-IIIb, HARMONIE Trial G1 Therapeutics Presents Preliminary Results from P-II Trial of Trilaciclib for Triple-Negative Breast Cancer at ESMO 2023 (..)

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European Pharmaceutical Review

MAY 22, 2023

The review stated that therapeutic innovation has resulted in the production of expensive drugs to treat rare diseases, which need to be available on time. Smart contracts, which are essential for easing international trade and logistics operations, can enable the user to track fake returns to the producer and supplier using RFID tags.

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pharmaphorum

JUNE 2, 2022

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. of the global population are affected by a rare disease.

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Pharmaceutical Technology

FEBRUARY 10, 2023

This is not the first treatment to come with a high price tag. However, more highly innovative/high-cost treatments, within infectious disease and neurology space, are likely to enter the market for rare disorders, increasing the number of non-oncology RSAs.

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FDA 105

Pharmaceutical Technology

JULY 14, 2022

It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Go-to-market strategies.

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Pharmaceutical Companies Pharmaceutical Companies Hospitals 59

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

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pharmaphorum

OCTOBER 21, 2021

Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug. Biogen stumped up $1.525 billion upfront for rights to the drug and another Sage candidate for neurological diseases last year, when the expectation was that Aduhelm was unlikely to get FDA approval.

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Insurance FDA 122

pharmaphorum

APRIL 5, 2022

Uveal melanoma is a rare cancer of the eye, which in the majority of patients is localised. mUM is the most common form of cancer affecting the eye in adults, but is still rare, diagnosed in a around 1,700 patients each year in the US, with a few hundred already with metastatic disease that has spread to other tissues.

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pharmaphorum

DECEMBER 12, 2022

Dutch biotech Argenx is on course to add another rare disease indication to the label of its FcRn blocker Vyvgart – primary immune thrombocytopenia (ITP) – thanks to new data reported at the ASH annual meeting.

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pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. million price tag. Zynteglo is already in beta-thalassemia in Europe, where the company will seek to expand access despite its hefty $1.8

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FDA 66

The Checkup by Singlecare

JANUARY 11, 2024

Read more about treatments for skin tags , hidradenitis suppurativa , and shingles. Growths “Growths such as cysts, skin tags, and moles can also cause pain when they’re inflamed,” says Brendan Camp, MD , a dermatologist with MDCS Dermatology in New York. Armpit pain can be managed with cold compresses, rest, and sometimes medication.

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pharmaphorum

MAY 17, 2021

C5 inhibitor Soliris – which has a list price in the US of more than $500,000 per year – has been a standard therapy for the rare disorder since 2007, and was joined on the market by longer-acting follow-up Ultomiris in 2018 which launched with a price tag of around $450,000 per year. billion and $1.1 billion and $1.1

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pharmaphorum

JUNE 2, 2021

The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people with the rare genetic disorder who might be eligible to receive it. Zolgensma was backed for type 1 SMA in draft guidance issued last month, even though the gene therapy’s £1.79

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The Checkup by Singlecare

MARCH 21, 2023

Opioids are prescription drug painkillers that are typically reserved for certain types of moderate to severe and sometimes chronic pain , and rarely for acute pain that is unresponsive to non-opioid medications like non-steroidal anti-inflammatory drugs ( NSAIDs ), Tylenol ( acetaminophen ), and certain antidepressants or anti-seizure medications.

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The FDA Law Blog

JUNE 27, 2023

Amongst his accomplishments, Law360 considered the role James has played in leveraging little-used pathways to FDA approval for often first-ever drugs to treat rare diseases (e.g., Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS). Dormer Jeffrey N. Gibbs Paul M. Hyman Alan M. Kirschenbaum Allyson B.

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FDA 81

pharmaphorum

AUGUST 26, 2022

BioMarin will no doubt have an eye on the earlier experience of bluebird bio, which secured EU approval for rare disease gene therapy Zynteglo (betibeglogene autotemcel) in 2019 for beta thalassaemia with a price tag of around €1.6 million spread over five years.

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pharmaphorum

AUGUST 10, 2020

Evrysdi is the third treatment approved for SMA , an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene abeparvovec). But Spinraza costs $750,000 in the first year of treatment and about half that price annually from then on.

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European Pharmaceutical Review

MAY 11, 2023

Rare, genetic diseases like SMA are prime targets for gene therapy, but by their very nature, early signs can be difficult to recognise, delaying diagnosis and treatment. 1 This is assuming a $1M price tag for all gene therapies. In reality, this will vary by disease area and therapy.

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FDA 70

Pharmaceutical Technology

JANUARY 27, 2023

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. A refund up to 12 inpatient doses will also be offered for patients whose disease worsens while receiving treatment.

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