BioMarin announced that its drug, Brineura (cerliponase alfa), received recommendation by NICE’s Highly Specialized Technology committee.
Pharma Analyst Kajal Jaddoo of GlobalData, a data and analytics company, offered her view on the potential the drug has. According to GlobalData, Brineura global sales are predicted to reach $358m by 2025.
“Brineura has the potential to be commercially successful as currently there is no cure or life-extending treatments for neuronal ceroid lipfuscinosis type 2 (CLN2), a rare inherited disease and GlobalData forecasts that Brineura will generate global sales of $358m by 2025.
“Though Brineura has received approval, it will still need to be evaluated by an ongoing judicial review and payers before it gains widespread use, meaning the drug may face a slow uptake.
“Brineura is a recombinant human tripeptidyl peptidase enzyme. It is administered by intracerebral route through patented technology. The drug was indicated to slow loss of movement ability in symptomatic pediatric patients aged three and older with infantile CLN2. Brineura is tripeptidyl Peptidase 1 (TPP1) replacement agent. It restores TPP1 enzyme activity and breaks down the storage materials that cause CLN2 disease.”
To read more about Brineura, you can read the comprehensive article we wrote on the drug here in May.
Fake Weight Loss Drugs: Growing Threat to Consumer Health
October 25th 2024In this episode of the Pharmaceutical Executive podcast, UpScriptHealth's Peter Ax, Founder and CEO, and George Jones, Chief Operations Officer, discuss the issue of counterfeit weight loss drugs, the potential health risks associated with them, increasing access to legitimate weight loss medications and more.
MDMA Therapy for Mental Health Conditions: Do the Benefits Outweigh the Risks?
October 25th 2024Despite a recent FDA Complete Response Letter issued to Lykos for midomafetamine capsules for the treatment of post-traumatic stress disorder, experts believe that the future is bright for psychedelic drugs that treat mental health conditions.
The Transformative Role of Medical Information in Customer Engagement
October 3rd 2024Stacey Fung, Head of Global Medical Information at Gilead Lifesciences, delves into the evolving role of Medical Information (MI) in the pharmaceutical industry. Covering key topics like patient engagement through omnichannel strategies, combating misinformation, and leveraging AI to enhance medical inquiries, the conversation with Stacey highlights MI's critical role in ensuring patient safety and supporting drug development. She also shares her professional journey and tidbits for early career professionals on professional development.
The Impact of Artificial Intelligence on the Creation of Medicines
October 24th 2024Najat Khan, chief R&D officer, chief commercial officer, Recursion, and Fred Hassan, director, Warburg Pincus, discuss how artificial intelligence can help reduce healthcare costs at the 20th Annual Young & Partners Pharmaceutical Executive Summit held at the Yale Club of New York.