Ultrgenyx Pharmaceutical, the Novato-based rare disease specialist is on fire. The company earned approvals for two different medications within six months from the Food and Drug Administration, a feat that Big Pharma companies four times the size of Ultragenyx can’t boast.
The company and its development partner Kyowa Hakko Kirin Co. Ltd. gained an approval for Crysvita, an injectable treatment for XLH, a rare disease. It causes rickets and lower body bone deformities in children as well as bone and dental pain and decreased height. In adults, the disease greatly increases the likelihood of fractures, causes joint pain, hearing loss, impaired movement and dental issues.
Crysvita had been given Orphan Disease as well as Breakthrough Therapy Designation by the FDA, which gave Ultragenyx certain development and tax advantages as well as eligibility for a Rare Pediatric Disease Priority Review Voucher.
All of that may sound a little too inside baseball, so allow me to explain. The breakthrough status is reserved for drugs that treat serious diseases that if approved will impact patients with respect to efficacy and safety of the treatment. Perhaps more importantly for the company, it also hands them the Review Voucher which can be used for other drugs in the company pipeline or can be sold.
The company received its first voucher last November when Mepsevii received its FDA approval. A month later it sold the voucher to Novartis for $130 million.
Ultragenyx estimates that the XLH market is worth about $1 billion a year.
Dillon Beach Resort sold
Dillion Beach Resort, the West Marin 55 acre property was owned by Nancy and Fred Kline of Cline Cellars and Jaccuzi Family Vineyards across the street in Sonoma Valley. But the resort was sold to a group led by San Anselmo resident Mike Goebel. Goebel owns Petaluma’s Brewster’s Beer Garden.
Locally the resort, which consists of a café, a trio of rental cottages, a general store and a park, which is known to locals as friendly to visitors who have four-legged friends. It’s seen by many in the dog community as an alternative to Point Reyes Seashore, which has stipulations that dogs can explore only three beaches and one trail as well as leash regulations, while Dillion welcomes behaved canines off-leash.
Rumors of the deal floated earlier this year and the resort was eventually priced at $7 million. The Cline’s purchased the property for $2.7 million in 2001, but said they wanted to part with it because they couldn’t give it the attention it deserved anymore. The purchase price paid by Goebel and company has not yet leaked out.
It is said that the new ownership plans to refurbish the cottages, shut down the store to give the boot to termites as well as rework the café menu in a bid to make Dillon Beach more of a community gathering spot.
Your Marin moment
Downtown San Rafael merchants are cheering that the city is doing a long hard review of the look of the Fourth and A Street intersection. Currently there are four concrete corner benches and liquid amber trees that are tearing up the sidewalk.
The pilot program checks in at $130K, but giving the intersection a fresh look isn’t a cheap undertaking. One factor is of course that process is king in Marin and eventually this will need the city council to give its blessing. But more than that the downtown is the center of the Mission City and change has always come hard to this valued real estate.
When Macy’s abandoned Fourth Street in 1996, the downtown was a collection of empty store fronts and retailers struggling to pull shoppers in. I caught plenty of flack for a story I wrote at the time, suggesting that one could fire a cannon down the main street and not hurt anyone.
Today, after a massive rebranding effort by the city as municipal construction, the area is a beehive of activity day and night, and parking can be challenging.
But issues still smolder. The homeless are a high-profile thorn in the city’s side, even for this project. Critics have wondered if changing out benches is a subtle effort to keep the homeless from getting too comfy on that corner.
But Economic Development Director Danielle O’Leary assures that is not the case. According to reporting in the ubiquitous Marin IJ, “detouring the homeless is not the driving force for this pilot seatwall design concept. The city has worked very hard to design a comprehensive and compassionate approach to our homeless services.”
In this case the substitution of “detouring” for “booting out” is a excellent example of bureaucratic language tossing a nice spin. The intersection isn’t the only thing the city is cleaning up.
