news
AstraZeneca agrees novel $1 billion endocrine acquisition
As part of the proposed acquisition, Alexion (AstraZeneca Rare Disease) will add a therapeutic to its pipeline that has potential to “shift the treatment paradigm” in hypoparathyroidism.
List view / Grid view
Rare diseases
article
Making ATMPs a reality for rare disease patients
Advanced therapy medicinal products (ATMPs) will be among the first to undergo joint clinical assessments at EU level in 2025 and these will form the basis for national value assessments and pricing negotiations. Here, Paolo Morgese from the Alliance for Regenerative Medicine discusses how methodologies for joint clinical assessments (JCAs)…
news
CHMP meeting highlights: February 2024
Ten new medicines, including a new oligonucleotide therapy for ALS, were recommended for approval at the Committee for Medicinal Products for Human Use (CHMP)’s recent meeting.
news
CHMP meeting highlights: January 2024
In its first meeting of 2024, the CHMP recommended a generic medicine for schizophrenia and refused a marketing authorisation (MA) for geographic atrophy.
news
Sanofi bets $1.7b on potential best-in-class rare disease therapy
Under its newly agreed acquisition, Sanofi is to acquire Inhibrx, Inc., adding INBRX-101 to its portfolio, a therapy that could improve the treatment options available for alpha-1 antitrypsin deficiency (AATD).
news
AstraZeneca to advance haematology cell therapy
The new acquisition grants AstraZeneca rights to a new CAR-T cell therapy with a differentiated manufacturing process that could provide a potential best-in-class blood cancer treatment.
video
mRNA therapies: a new frontier
Nicolas Chornet, Senior Vice President of International Manufacturing at Moderna, speaks to EPR about the future of mRNA therapies and offers an update on the company’s international expansion plans.
news
MSD medicine accepted for use in Scotland
Scotland is the first European country to accept the HIF-2α inhibitor WELIREG® (belzutifan) for eligible adults with von Hippel-Lindau (VHL) disease.
article
Exploring the gene therapy biomanufacturing landscape
In this exclusive Q&A with European Pharmaceutical Review, Sheila Ann Mikhail, co-founder of AskBio and keynote speaker at CPHI Barcelona delves into the current landscape and future potential of the gene therapy sector. She explores strategies for making gene therapies more affordable and accessible, innovations in drug delivery, as well…
news
Small molecule drug shows promise in rare disease
In a Phase II trial, the small-molecule drug CBL-514 demonstrated significant reduction in lipoma size and pain improvement in Dercum's disease, a rare disorder.
news
CHMP meeting highlights – May 2023
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.
news
$1.7b acquisition to develop rare haematology therapy
When CTI BioPharma is acquired by Swedish Orphan Biovitrum AB (Sobi), the companies intend to advance an oral kinase inhibitor treatment for a rare haematological disease.
news
Takeda to build £600m plasma therapy manufacturing site
Takeda’s largest manufacturing capacity expansion investment in Japan will fund the build of a new facility for plasma-derived therapies.
news
CHMP recommends first pegylated enzyme for Fabry disease
A PEGylated enzyme replacement therapy for Fabry disease has received recommendation for a marketing authorisation from the European Medicines Agency (EMA)'s human medicines committee.
news
mRNA non-vaccine therapeutics to generate $2 billion by 2028
Driven by the approval of pipeline agents, mRNA non-vaccine therapeutics are set to reach $2 billion in sales by 2028, says GlobalData.
