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Orphan drugs face “uphill battle” in 2020

A new analysis from GlobalData has claimed that the number of orphan drug designations (ODD) being handed out by both the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) is declining.

The company has revealed that over the last six years, the numbers have slowed down despite the visible benefits of such designations in speeding these drugs through development and to patients.

Quentin Horgan, pharma analyst at the firm explains that the EMA had a “much greater” decline in unique drugs awarded ODDs than the FDA - with the EU-based organisation showing a 49% decrease between 2014 and 2019, while the FDA only had an 8% decrease across the same time period.

He went on to say that the FDA decline is “however, more surprising considering that 2017 was a bumper year for drugs awarded ODDs in the US, significantly beating previous records.

“This large spike in 2017 for the US could be attributed to the FDA’s Orphan Drug Modernisation Plan, which began in 2017 with the aim to clear out the backlog and streamline the ODD application process.”

The outlet says that its database shows that a third of all pipeline drugs are indicated for rare diseases - ultimately showing that despite ODDs being on the decline, the pharmaceutical industry’s “enthusiasm for these niche drugs is still going strong.”

Earlier this week, Janssen and MeiraGTx’s novel AAV-RPGR drug for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP) - a therapy that was designated Orphan Drug back in 2016 - was handed Priority Medicines (PRIME) and advanced therapy medicinal products (ATMPs) status.

ODD is a status awarded to drugs that intended for the treatment, prevention or diagnosis of a rare disease or condition, which affects less than one in 200,000 people. The system makes sure that the candidate receives government funding to enable a company to further development.

5th March 2020


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