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.

Marketing 105

Marketing Medicine Leads Safety 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.

Medicine 59

Medicine Marketing Specialization Pharma 59

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.

Side effects 52

Side effects Sales Competition Patients 52

PM360

APRIL 22, 2024

In the swiftly changing realm of advanced therapies like biosimilars, rare disease molecules, and cell and gene therapies, a strong scientific interpretation and orientation is essential. Teams are stepping up to this challenge, actively shaping the trajectory of new interventions right from their inception.

Medical 52

Medical Healthcare Healthcare Education 52

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.

Patients 105

Patients Pharma Education Doctors 105

PM360

APRIL 7, 2023

The shift toward longer, more adequate timeframes to establish proof of concept can be as long as three to four years, 1 given the complex requirements for developing and launching specialty drugs and cell and gene therapies (CGTs) for individuals with complex conditions or rare or orphan diseases.

Manufacturing 52

Manufacturing Marketing Patients Safety 52

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.

FDA 109

FDA Marketing Competition Patients 109

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.

Patients 52

Patients Pharmaceutical Manufacturing Healthcare Provider 52

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.

Food and Drug Administration 68

Food and Drug Administration Government Healthcare Healthcare 68