Raptiva

1
Raptiva (efalizumab) BenefitRisk Assessment

• Charles Johnson, MB, ChB
• Senior DirectorHead of Specialty Biotherapeutics
• Genentech, Inc.

2
A Significant Unmet Medical Need Exists

• Psoriasis is a chronic life-long disease with
  QOL impact similar to other major
  diseases(arthritis, diabetes, cancer,
  cardiovascular disease)
• Common physical symptoms of chronic psoriasis
  include skin pain, itching, and bleeding these
  physical symptoms impair quality of life
• Topical medications are insufficient to treat
  moderate to severe disease
• Currently approved therapies for moderate to
  severe disease are effective, but have
  significant safety limitations and accessibility
  issues

3
Characteristics of Raptiva in Psoriasis

• Dosed once weekly as a single SC injection
• Has an early onset of action
• Effective at 12 weeks
• 27 of patients achieve PASI-75 (12 weeks)
• 59 of patients achieve PASI-50 (12 weeks)
• Extended treatment improves response at 24 weeks
• 44 of patients achieve PASI-75 (24 weeks)
• 66 of patients achieve PASI-50 (24 weeks)
• Response maintained with one year continuous
  therapy
• 45 of patients achieve PASI-75
• Therefore best used as continuous therapy
• Safe and well tolerated

4
Raptiva Improves Plaque Psoriasis
Before Treatment
After Treatment
Day 0 PASI 50.4
Day 91 PASI 2.1 PGA Excellent
5
Quality of Life Improves Quickly
Study 2390
Hierarchical rank-sum test, Raptiva vs. placebo
6
Key Safety Outcomes

• Extensive safety database 2762 treated patients
• Most common adverse events are mild flu-like
  symptoms following first 2 Raptiva injections
• Favorable overall adverse event profile,
  including infection and malignancy
• Psoriasis adverse events are infrequent and
  manageable
• Infrequent cases of reversible thrombocytopenia
• No evidence of renal or hepatic dysfunction

7
Raptiva is Safe and Effective

• Meaningful clinical benefit has been demonstrated
  in patients with moderate-to-severe plaque
  psoriasis
• Ongoing therapy with Raptiva provides extended
  benefit, with no increase in adverse events as
  exposure is prolonged
• Frequency of psoriasis adverse events is
  mitigated by
• Continued therapy in responders
• Limiting dose to 1 mg/kg
• Transition in non-responders (12 weeks)

8
BenefitRisk Conclusions

• Based on the robust efficacy and reasonable
  safety, Raptiva should be available as a
  therapeutic option for patients with moderate to
  severe plaque psoriasis
• Post approval surveillance will further clarify
  the long term safety of Raptiva

9
Raptiva Proposed Indication

• Raptiva is indicated for the treatment of adult
  patients (18 years or older) with moderate to
  severe plaque psoriasis.