Washington Editor

Shire Human Genetic Therapies (HGT) is getting social as it launches its newly approved Firazyr, a self-administered injectable treatment for acute attacks of hereditary angioedema (HAE) in adults.

The company's marketing model for Firazyr (icatibant), which was approved by the FDA Aug. 25, will focus on patient services and connecting with patients.

At the center of the marketing is Shire's OnePath website, a one-stop resource for doctors and patients, said Sue Bruhn, senior vice president of strategic planning and program management at Shire HGT, a division of Dublin, Ireland-based Shire plc.

In addition to providing information about the diseases Shire HGT is addressing, the site provides patient assistance programs, case managers, training programs for self-administration and other resources.

HAE, a genetic disorder caused by low levels or the improper function of the C1 inhibitor protein, affects fewer than 30,000 people in the U.S., according to the FDA.

People with HAE can experience rapid swelling of the hands, feet, limbs, face, intestinal tract, voice box or windpipe, which could result in disfigurement, disability or death.

Patients with the condition have a close-knit online community with an active organization, the HAE Association. Shire HGT tapped into that community in developing Firazyr for the U.S. market.

For instance, the Lexington, Mass.-based company held focus groups to design educational materials and ensure packaging would be easy to use if a patient's hands were swollen during an acute attack, Bruhn told BioWorld International.

The company used a similar strategy in its market research for pricing. It met with a number of payers before setting the price at $6,800 per treatment, according to Hugh Cole, HAE global franchise leader at Shire HGT.

"The product brings a great deal of value to the patient," Cole told BioWorld International.

Payers recognize that value and appreciate that self-administration of the drug could eliminate costly trips to an emergency room, he added.

Since the severity and frequency of acute attacks are unpredictable, it's difficult to know the annual treatment cost per patient. Because the prefilled syringes are intended for emergency use, they can be stored at room temperature for up to 18 months.

Several products have been approved in the U.S. for prophylactic use in HAE, and Dyax Corp.'s Kalbitor (ecallantide) was approved for acute HAE attacks in late 2009. But Firazyr is the first self-administered drug approved for acute breakthrough attacks, Bruhn said. A patient's only option before was to "tough it out" or go to the emergency room for treatment.

Having an on-demand treatment will give patients power over the disease, Bruhn added.

Firazyr is already approved in Europe, and it got the OK for self-administration there a few months ago. Its approval path in the U.S. was a bit bumpier. The drug was originally developed by Jerini AG, using two pivotal trials designed for European approval, Bruhn said.

One trial compared Firazyr to the European standard of care, which is not approved in the U.S. The other was a placebo-controlled trial that missed its endpoint, Bruhn said. When Jerini filed for approval in the U.S., it received a not approvable letter in 2008.

Shire bought Jerini later that year in a cash deal valuing the German firm at $519 million to acquire Firazyr, knowing it would have to conduct an additional Phase III trial. It reported positive efficacy and safety results from that study last December.

Other Drugs in the Neighborhood

Other biotechs in the HAE community welcomed their new neighbor. "It's going to be a great drug for acute patients," Will Roberts, ViroPharma Inc.'s vice president of corporate communications and investor relations, told BioWorld International. The Exton, Pa.-based company makes Cinryze (C1 Esterase Inhibitor [human]), which is approved in the U.S. as a self-administered prophylactic HAE treatment for adults and adolescents.

Having a new voice in the community will help in communicating the severity of HAE and raising awareness of the disease, Roberts said.

Although Cinryze will not compete with Firazyr in the U.S., the drugs may soon be competing in Europe. The ViroPharma drug was recently approved in Europe with a broader label that includes prevention, treatment of acute attacks and pre-procedure use, Roberts said. Cinryze will be launched in Europe later this year.

Recently, Europe health authorities also approved an expanded label allowing for self-administration, by intravenous infusion, of CSL Behring's Berinert to treat acute HAE attacks.

The Berlin-based company's C1-esterase inhibitor was approved by the FDA in 2009 for the treatment of acute abdominal or facial attacks of HAE in adolescent and adult patients. However, its U.S. label doesn't include self-administration.

Earlier this month, Pharming Group NV, of Leiden, the Netherlands, got the go-ahead to start treating patients in a U.S. Phase IIIb study of Rhucin, a recombinant human C1 inhibitor manufactured from the milk of transgenic rabbits, as a treatment for acute HAE attacks.

Pharming and its partner Santarus Inc., of San Diego, received a refusal-to-file notice from the FDA earlier this year citing insufficient data to support the proposed dose.

The new trial is being conducted under a special protocol assessment and is expected to be completed by the third quarter of 2012. Rhucin, marketed as Ruconest, was launched in Europe last December.