Hyalcis film, tested on animal models (rat and sheep) revealed significant benefits that were recognized by the EMA when the orphan designation was given:
1) Effectiveness. The cisplatin/hyaluronate film applied on the animal chest had two advantages: i) due to the local cavity absorption, the drug exposure of pleura mesothelium greatly increased, ii) drug clearance was slowed down.
2) Low toxicity. A preclinical pharmacokinetic and toxicity study has been carried out on sheep. Also the loco-regional drug administration of cisplatin solution reduced the toxic effects in MPM patients, compared to i.v. chemotherapy but the effectiveness was low.
3) Safer and very efficient drug administration. The sheep study has been aimed also to test in large animals the pleural application of cisplatin/hyaluronate film, in order to assess the film handle ability in surgical practice, drug release and bioavailability. The film application had no critical risks for surgery personnel, unlike the intrapleural cisplatin solution that could leak from the cavity, due to the pressure applied during surgical suture.
On 29 August 2016, the Orphan Medicinal Product designation (EU/3/16/1719) was granted by the European Commission to PlumeStars, for the cisplatin/hyaluronate film, named Hyalcis, for local treatment of MPM. Hyalcis is an intracavitary film to stuck, during tumour surgery, on lung pleura to prevent local MPM metastases. The designation of orphan medicine has also been obtained by US FDA.
The orphan designation entitles PlumeStars as unique owner of Hyalcis product. Thus, it is a company value and offers business perspectives that PlumeStars intends to exploit. In details, no similar product for the local treatment of mesothelioma recurrences exists on the market. Starting from the registration date, Hyalcis marketing authorization will give to PlumeStars 10 years of market exclusivity in Europe (7 years in USA). More than 43,000 people die each year worldwide for mesothelioma and 10,000 new cases in Europe, North America, occur each year. The value of mesothelioma market is estimated at over 600 M Euro. PlumeStars intends to reach 28% of this market within 8 years from the end of the SME Phase 2 project (see Section 2). The market dimension could expand beyond mesothelioma treatment, since this medicine can be proposed for other sensitive cancers surgically treated, such as the ovarian or peritoneum cancers. Thus, an inviting opportunity for creating new jobs for the manufacturing line of new film products and larger market is present.
