Reuters Puts Orphan Drugs Among Top ‘Ones to Watch’

In Partnering & Investment by Cameron

Reuters Ones to Watch

A Report published this month by Reuters has placed a number of orphan drugs among their top picks for 2014. The report looks at their top five picks from among recently launched or approved drugs, as well as the different clinical phases.

The report cites the maturing of the FDA’s orphan drug designation program, now over 30 years old, and the EMA’s orphan medicinal product staus as the reason behind the growing success of orphan drugs across the industry. So what orphan drugs made the short list?

Already launched or receiving approval


Topping the list is the Chronic Lymphocytic Leukemia (CLL) treatment produced in partnership by Gentech (Biogen Idec) and Chugai Pharmaceutical (Roche). Gazyva (obinutuzumab) is indicated for use in conjunction with chlorambucil, for patients with previously untreated CLL. Gazyva wobks by binding to CD20, an activated-glycosylated phosphoprotein target expressed on the surface of all B-cells, causing cell death by apoptosis. The treatment was granted breakthrough therapy designation as well as orphan drug designation by the FDA back in November 2013. there is also currently a Marketing authorisation Application (MAA) currently out to the EMA for the treatment.

Reuters estimates that the Cazyva will earn $166 million in global revenue this year, rising to $1.1 billion by 2017.

In preclinical trials, Gazyva showed enhanced antibody-dependent cell-mediated cytotoxicity and lower complement recruitment compared with first-generation antibodies. In a phase III trial (NCT01010061), Gayzva plus chlorambucil improved progression-free survival, complete response rates, overall survival and reduced the risk of disease worsening or death by 61 percent, compared with Rituxan plus chlorambucil


Also launched last quarter was Ceptaris Therapeutic’s (Actelion) stage IA and IB mycosis fungoides (MF)-type cutaneous T-cell lymphoma (CTCL) treatment Valchlor. Valchlor (mechlorethamine) is a topical gel formulation of the nitrogen mustard cytotoxic alkylating agent mechlorethamine, and is indicated for use with patients who have already undergone some sort of skin-directed therapy. Branded as Mustargen, Ceptaris have been marketing the product as an intravenous treatment for Hodgkin disease, NHL and leukemias and off-label in gel or ointment formulations to control mycosis fungoides.
for several decades.

Reuters predicts that global sales will reach $96 million a year by 2019.


Back in October Actelion’s Pulmonary arterial hypertension (PAH) treatment, Opsumit. Opsumit (macitentan) replaces Actelion’s blockbuster Tracleer (bosentan), which recorded sales of 1.5 billion francs in 2012, and is set to go off patent in 2015. This oral tissue-targeting endothelin-A and -B receptor  antagonist has recieved orphan drug designation from both the FDA and EU and improves upon Tracleer in a number of areas. Offering a convenient one-a-day dosing, improved safety and no need for continuous liver testing Opsumit looks set to outpace Tracleer and the number of generics set to hit the market later this year.

Reuters predicts that sales of Opsumit will hit $1.1 billion a year by 2017.

Promising Phase I entries


In December Xentec Biosciences’ PulmoXen, a treatment for Cystic fibrosis, entered into a Phase I safety trial to assess the tolerability of the substance in healthy volunteers.

PulmoXen is a polysialylated formulation of recombinant human DNase I, an enzyme which cleaves extracellular DNA thereby reducing the viscosity of purulent and infected lung secretions such as the sputum of cystic fibrosis patients. The agent is delivered using Xenetic’s PolyXen delivery technology which incorporates bacterial polysialic acid to increase the active life and improve pharmacokinetics and pharmacodynamics of peptides, proteins and small molecule drugs.

In preclinical studies, the technology has been shown to preserve drug functionality when conjugated, improve in vivo stability, prolong pharmacological action and active life of drug in blood, reduce frequency and volume of dosage, and reduce immunogenicity and antigenicity.

PilmoXen is set to replace Roche’s Pulmozyme with the benefit of reduced dosing for patients.

To get the full reuters report on over 800 drugs click here.