Orphan diseases review

Lysosomal Storage Disease

Deals Review of Lysosomal Storage Diseases and Other Orphan Indications

Deals coverage on Cortellis Competitive Intelligence indicates an increasing trend in the number of deals that have been forged in the area of lysosomal storage diseases (LSDs) and other orphan indications. However, it is not clear exactly how many as numerous existing drugs have been assessed for new orphan indications later in their development stages. The following article reviews the licensing portfolio of a number of high revenue-generating LSD/other orphan indication treatments on the market, as well as certain significant and promising therapeutic candidates within the field, as featured in Cortellis Competitive Intelligence.

Protalix’s Elelyso the Subject of Pfizer’s $115 Million Interest

Protalix’s glucosylceramidase stimulator, Elelyso, was the subject of a $115 million agreement involving Pfizer. Following recent US fast-track and orphan drug statuses, and US filing for Gaucher disease type I whilst in advanced clinical development for other major territories, Pfizer identified the enzyme replacement therapy (ERT) as a profitable one (with expected sales of $275 million in 2019) and acquired exclusive worldwide rights (excluding commercialization in Israel) in November 2009. Protalix received million upfront with million in regulatory milestones expected. The following month, Protalix received a million milestone for the completion of a rolling NDA submission, and submission of a PIP to the EMEA’s pediatric committee for a trial. In June 2012, a million milestone was triggered by FDA approval a month earlier, however, at the time, the CHMP recommended against EU approval, due to approval of Shire’s orphan drug designated Gaucher treatment, Vpriv, in August 2010.figure 1 After EU disapproval in November 2012, an amendment a month later meant that Protalix would continue to manage, administer and sponsor ongoing trials while Pfizer would conduct and sponsor new trials. Protalix could receive $8.3 million if a clinical milestone was reached. Deciding against marketing in Brazil, Pfizer returned rights to Protalix in January 2013 in return for a maximum of approximately $12.5 million from Protalix’s net profits in Brazil per year bringing the current value of the deal to $135.8 million.

Figure 1. Timeline of major events for Protalix and Pfizer’s agreement for Elelyso

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