Yesterday, OPKO Health, Inc. (NYSE: OPK) announced that the company received a Complete Response Letter from FDA for Rayaldee™ new drug application.  It appears that FDA did not cite any safety efficacy or labeling issues and no additional studies were requested to be conducted prior to FDA approval and the company continues to work with FDA and their third party manufacturer to resolve any outstanding issues.  Rayaldee™ is a treatment for Secondary hyperparathyroidism (SHPT)  in stage 3-4 CKD patients with vitamin D insufficiency.  The condition occurs when the parathyroid glands secrete excessive amounts of parathyroid hormone and there are insufficient quantities of vitamin D hormones to maintain a state of balance between calcium and phosphorus in the body.  This imbalance may lead to excessive calcium to be released from bone leading to weakening of the bones and calcification of vascular tissues.  Last year, The National Kidney Foundation issued a clinical update stating that 40-80% of  patients with stage 3 or 4 CKD may suffer from the condition.

I’m excited to see Rayaldee™ become available to patients because we have been involved in the development of the product for many years.  We initially invested in Cytochroma, the company that developed Rayaldee™ before it was acquired by Opko Health.  When I joined BDC in 2007, Cytochroma was developing another novel compound to treat SHPT in CKD patients undergoing dialysis.  Having encountered some program delays and with changes in reimbursement policies for dialysis patients, the company, had to pivot and align around Rayaldee™.  With management’s expertise and support from the investors, Cytochroma was able to advance development into Phase III and attract the attention of Opko.

Bringing a product like Rayaldee™ through the clinical and regulatory process for drug development is the result of a lot of talent, a lot of capital, an awareness of the patient, prescriber and payer needs and thoughtful consideration of strategic options over an extended period of time.  As is often the case in biotech and specialty pharma start-ups, access to talent, capital and technology can be challenging.  While Canadian healthcare companies have access to world class technology, access to seasoned entrepreneurs and capital can be difficult.  More often than not, Canadian companies function with gaps in talent and experience at the senior levels and with lean financings that reduce the number of avenues available as companies need to consider strategic options. This makes the already perilous navigation from discovery to market even more daunting and risky for management and investors alike.  Innovative products like Rayaldee™, a first-in-class oral vitamin D prohormone treatment, expected to address tens of millions patients with stage 3 or 4 CKD, SHPT and vitamin D insufficiency, have the potential to make a difference in patient outcomes and we hope to soon see the Rayaldee™ product approved and launched making it available to patients.

Ela Borenstein

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