Four months after AstraZeneca received FDA approval for vandetanib, a treatment for inoperable medullary thyroid cancer, the company announced the drug’s trade name: Caprelsa. FDA said other proposed names – Zactima, for example – too closely resembled other currently marketed proprietary names.
Getting FDA approval on a brand name for vandetanib, AstraZeneca’s very first orphan drug, isn’t the only challenge the company has faced during launch. The size of the patient population eligible for Caprelsa is decidedly small, but ultimately fuzzy. “We don’t really know how many patients there are at any given time,” says Eric Vogel, executive director of oncology, at AstraZeneca. “In some reports its 1,000 [in the US], and the FDA thinks it may be 2,000 to 2,500.” Compounding the difficulties presented by a tiny patient population, relatively speaking, is the fact that not all medullary thyroid cancer patients would benefit from Caprelsa; the drug is indicated only for patients whose cancer “has progressed to the point where surgery is no longer an option.”
Vogel says the company is “learning as we go” during the launch. “We believe that roughly half of the patient population will end up in the major treatment centers around the country, but that the other half may or may not continue to be followed by the community endocrinologist or medical oncologist,” says Vogel. Traditional market research hasn’t uncovered a network of physicians treating medullary thyroid cancer, since Caprelsa represents the first pharmaceutical treatment option for those patients, says Vogel. “There are some things that we can do to find physicians that look like they might be treating [medullary thyroid cancer], but it’s an inexact science at this point.”
AstraZeneca signed an exclusive distribution deal with Biologics, an oncology management company and specialty pharmacy, last April. Biologics also distributes AstraZeneca’s Arimidex, as well as Novartis’ Afinitor, Gleevec and Tasigna, Bayer/Onyx’s Nexavar, Celgene’s Revlimid, BMS/Otsuka’s Sprycel, Pfizer’s Stutent, Merck’s Zolinza, Roche/Genentech’s Xeloda and others, according to an “in stock” list on the company’s website. The decision to partner with Biologics for distribution had to do with the size of the patient population, and the amount of support that patients using Caprelsa would need, says Vogel. “We’re pleased with the relationship. [Biologics] spends 45 minutes, on average, with each patient, according to the reports we get from them,” Vogel says. Biologics, for its part, provided a team of 10 nurse liaisons tasked with educating physicians about the drug, and easing the administrative burden on the physician’s practice, Dan Duffy, executive VP and general manager, oncology pharmacy services group, told PharmExec.
In addition to Biologics’ nurse liaisons, AstraZeneca has deployed “regional scientific managers” – physician and/or nurse-facing individuals, to “communicate to our HCP audience about risks associated with Caprelsa, the REMS programs and all of the precautions, as well as any questions they might have around the efficacy or safety of the product,” says Vogel. The REMS program has helped AstraZeneca identify customers, since physicians have to become certified through REMS before prescribing the drug. “Down the road, we may broaden our reach out to community oncologists, those who we’re already calling on with our sales forces for Faslodex,” says Vogel.
The company hopes new indications will be forthcoming, and is currently looking to Europe for the next Caprelsa launch. Laura Woodin, senior manager, corporate affairs, said in an email that Caprelsa is being evaluated in “more than 40 early-stage studies” and various tumor types, including pancreatic, glioblastoma (brain), biliary tract (liver duct), as well as two other forms of thyroid cancer, papillary and follicular. “We’re trying to take our learnings from the US and apply those to other markets,” says Vogel. In terms of a possible launch in the EU, Vogel says the patient population size across Europe is roughly the same as the US patient size, but right now, “it’s not efficient to commercialize [Caprelsa] country by country.” “We will commercialize it country by country as we get new indications, but we have to look at it more broadly by much larger markets” for now, says Vogel.
Caprelsa is currently under review with the European Medicines Agency (EMA) and Health Canada, according to Woodin. blog.pharmexec.com
As the number of drugs targeting small populations increases, managing numerous drug distribution channels and educating the relevant parties – specialty pharmacists, payers, physicians, and patients – becomes a daunting task. Specialty pharmacies that handle seven to 10 major disease states, after all, might only see three or four patients with a given rare disease, all year long.
To expedite distribution, and to make sure educational initiatives – under Risk Evaluation and Mitigation Strategies (REMS) requirements, and otherwise – are being met, AstraZeneca signed a deal with Biologics, an oncology management company, to distribute vandetanib exclusively through Biologics’ specialty pharmacy. Financial terms of the deal were not disclosed.
Approved in the US on April 6, vandetanib is an orphan drug indicated for the treatment of medullary thyroid cancer that can’t be removed by surgery, or that has spread to other parts of the body. In the US, somewhere between 500 and 1,000 patients, approximately, have this rare form of cancer, according to Dan Duffy, executive VP and general manager, oncology pharmacy services group, at Biologics. FDA puts the number at 1,300 to 2,200 in 2010, according to a release.
Biologics is tasked with managing vandetanib’s REMS requirements (only REMS-certified pharmacists are allowed to dispense the drug) in addition to working with payers and consolidating referral sources. Ten nurse liaisons are “going out to educate the physician and clinical staff about program specifics, how to make it efficient for them, and to ease the administrative burden for the physician,” said Duffy. For patients, the company expedites available copay assistance, with a focus on rapid turn-around times. Without insurance, a 30-day supply of 300 mg vandetanib, taken once daily, costs $10,454.00, according to an employee in Biologics specialty pharmacy. Patients without insurance may be elligible to recieve the drug at no cost, through AstraZeneca’s prescription savings program, according to Laura Wooden, a spokesperson at AstraZeneca. In clinical trials, vandetanib was “shown to affect the electrical activity of the heart, which in some cases can cause irregular heart beats that could lead to death,” hence the REMS, said FDA, in the release.
Wooden said in an email that the company’s exclusive distribution arrangement with Biologics is “a new approach for our US business…vandetanib is also the first treatment that AstraZeneca has developed and brought to market under orphan drug designation in the US.” Wooden said the small patient population for vandetanib was a major factor in signing the exclusive distribution deal. Duffy said the agreement lets AstraZenca “standardize the distribution channel and really control it.”
Vandetanib doesn’t currently have a brand name; AstraZeneca requested Zactima, but FDA did not accept that name, said Wooden. The company is currently in talks with FDA about a new name. blog.pharmexec.com
treatment aimed at ovarian cancer has been designated an orphan drug by the Food and Drug Administration. This means the drug, aimed at a disease that fewer than 200,000 people in the country get feeds.bignewsnetwork.com
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An orphan drug originally used for HIV treatment has been found to short-circuit the process that results in additional sensitivity and pain from opioid use. The study by researchers at the Indiana University School of Medicine is reported in the March 25, 2011 issue of Brain, Behavior and Immunity.
Source: Indiana University School of Medicine - Discipline: Physiology www.labspaces.net
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Italian biotechnology company, MolMed S.p.A., is expanding clinical trials of their new drug NGR-hTNF to Europe and the US. Currently being tested in ten Italian facilities, NGR-hTNF has been developed for treatment of pleural mesothelioma. NGR-hTNF has been designated as an ‘orphan drug’ in the US, which means its target disease is an ‘orphan disease,’ affecting less than 200,000 people at a time.
Mesothelioma is a rare cancer of the abdominal cavity lining. Pleural mesothelioma is specific to mesothelioma of the lung lining, while peritoneal mesothelioma is that of other abdominal linings, such as the heart. Pleural mesothelioma is the more common of the two, affecting about three thousand Americans each year and an estimated ninety thousand worldwide.
Mesothelioma is caused by asbestos exposure. Asbestos fibers and dust can begin a mutative development if inhaled or ingested. Often taking twenty to fifty years to show signs and symptoms, mesothelioma grows irregularly patterned malignant tumors through the affected area. Many mesothelioma patients have no idea they were ever exposed to asbestos, making diagnosis difficult. Symptoms mimic those of pneumonia and bronchitis, so once patients do seek a physician’s counsel, proper diagnosis can be further postponed.
There is no known cure for mesothelioma. Many treatments are used to make patients as comfortable as possible and to extend life expectancy, which currently averages eighteen months following diagnosis. Mesothelioma treatments may include surgery, chemotherapy, radiation therapy and pain management.
Three hundred and ninety patients are expected to participate in the upcoming NGR-hTNF clinical trials. All patients have been diagnosed with malignant pleural mesothelioma and have undergone at least one round of treatment using the current standard pemetrexed-based chemotherapy. These new studies will focus on patients quality of life, survival times, progression-free times, and disease control in response to NGR-hTNF. Not only will the results of the clinical trials be a welcome addition in the medical community, but the studies will allow many mesothelioma patients to receive new, cutting edge treatments. Results are expected in 2013.
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