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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

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Psychedelic medicines: are they gaining traction in Europe?

European Pharmaceutical Review

As psychedelics diffused into wider society and recreational use increased, some individuals reported…ongoing symptoms after the drugs had left the body” At a time when psychiatry lacked effective medical therapies, the discovery of LSD was of interest.

Medicine 118
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FDA Awards HistoSonics Clearance of its First-of-a-Kind Edison® Histotripsy System

Legacy MEDSearch

Food and Drug Administration’s (FDA) De Novo Classification Request process, a rigorous pre-market review pathway for medical devices with no existing predicate. Marketing authorization makes Edison the first and only histotripsy platform available in the Unites States.

FDA 52
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Regulatory-first for gene editing therapy

European Pharmaceutical Review

The Investigational New Drug (IND) application for Intellia Therapeutics’ in vivo CRISPR-based candidate NTLA-2001, has been cleared as a gene editing therapy for transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) in the US. This supports NTLA-2001’s potential as a single-administration therapeutic.

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New quality requirements for tobacco products

European Pharmaceutical Review

2 The US Food and Drug Administration (FDA) has recently announced that it will control the quality of tobacco products, particularly e-cigarettes, more closely, to prevent avoidable contamination and help address “inconsistencies between product labelling and actual concentrations” in these products, potentially misleading customers.

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Reality Bites: Disappointment in the R&D Sector

Pharmaceutical Technology

As the blockbuster drugs of the 90s that earned the industry billions reach their patent shelf lives, pharmaceutical companies require new medicines to sustain an estimated $157bn worth of sales. Even so, current figures show firms’ biggest portion of spend is still on marketing and administration at 33.1%,

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A history of blood cancer treatment

pharmaphorum

2001– FDA green lights revolutionary treatments. Just over a decade after it was developed by biochemist Nicholas Lyndon, Imatinib received US Food and Drug Administration (FDA) approval in 2001. Today, the bone marrow donor registry comprises more than 39,527,166 donors and 804,246 cord blood units.