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Novartis releases new data for innovative rare kidney disease treatment
Data from the pre-specified interim analysis of Fabhalta® (iptacopan) indicate a statistically significant reduction in proteinuria reduction.
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mRNA therapy could provide intracellular protein replacement for rare disease
With no treatments approved for propionic acidaemia, Moderna’s LNP-encapsulated mRNA therapy could offer a new therapeutic option for patients, new data suggests.
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CHMP meeting highlights: March 2024
At the Committee for Medicinal Products for Human Use (CHMP)’s March meeting, twelve medicines received positive opinions for marketing authorisations, including a new antibiotic and a novel oral medicine for a rare blood disorder.
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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.
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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)…
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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.
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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.
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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).
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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.
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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.
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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.
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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…
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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.
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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.
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$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.
