By Ben Adams
|MedImmune's Bing Yao|
The FDA has given AstraZeneca's ($AZN) biologics arm MedImmune the thumbs up for an orphan drug status for its rare disease treatment--meaning the biotech may at last soon start to make a dent into the $15 billion paid for it nearly a decade ago.
MedImmune's MEDI-551 is currently in mid- to late-stage trials for neuromyelitis optica (NMO)--a rare autoimmune disease that can be fatal.
The orphan status is a coveted allowance as it gives greater patent protection for new medicines that can help patients with an unmet medical need.
MEDI-551 is a humanized, monoclonal antibody that binds with high affinity to CD19, a protein expressed on a broad range of B cells, including certain B cells called plasmablasts. MedImmune is also studying the drug in a mid-stage trial for patients with diffuse B-cell lymphoma.
In NMO, immune system cells and antibodies attack cells in the optic nerves and spinal cord, with its prevalence estimated to be around 5 out of 100,000 people. It primarily affects women and it may be more common in non-Caucasians. There is currently no cure or approved treatment for NMO.
MedImmune is currently enrolling several hundred patients into a late-stage, multicenter, multinational, randomized, double-masked, placebo controlled study with an open-label extension period to evaluate the efficacy and safety of the drug. The study is expected to report its first data sets in 2018 and be completed in 2019.
Bing Yao, senior vice president of R&D and head of the Respiratory, Inflammation and Autoimmunity Innovative Medicines unit at MedImmune, said: "The orphan designation for MEDI-551 underscores the significant need for an effective medicine for NMO, a rare, devastating disease which causes increasing damage and disability with each attack. MEDI-551 has a unique, targeted mechanism of action offering potential for the treatment of NMO.
"We look forward to working with the FDA to advance MEDI-551 to patients suffering from NMO as quickly as possible."
MedImmune was bought by AZ in 2007 for an eye-watering fee of more than $15 billion--an amount criticized at the time as the Maryland-based biotech had only a limited number of early-stage pipeline drugs that typically focused around slow-growing flu vaccines and a respiratory drug for children, which turned out to not be so great.
AZ said it had its eyes on the future with its purchase but nearly ten years later, little justification has been made for the deal.
And in recent years the biotech has to shed hundreds of jobs in California as part of a major restructuring drive for its parent company--a company that has seen its fair share of late-stage disappointments and big changes at the top of its leadership since the MedImmune deal.
The biggest victim of AZ's failures was in fact its former CEO David Brennan, the man who signed off on the $15 billion-plus price tag for MedImmune--a decision that many believe was the main factor for his axing in 2012.
But since then, MedImmune has racked up the deals and partnerships with other firms and academic institutions--notably in cancer and rare diseases--and this orphan drug status for its new NMO drug is on reflection the most promising thing to have happened for one of its pipeline drugs in years.
It's likely not to be seeking marketing approval until the end of the decade given the three-year span of its current trial, but its additional studies for blood cancer could just prove to be the first real tangible revenue producer from the biotech firm. AZ will certainly be hoping so.