Pharmacology/Part 10/10.6 Biologics & Biosimilars

10.6 Biologics & Biosimilars

Biologics are complex molecules produced by living organisms, including antibodies, vaccines, and gene therapies. Biosimilars are highly similar versions of approved biologics.

Monoclonal Antibodies

Nomenclature

  • -omab: Mouse antibody
  • -ximab: Chimeric (human-mouse hybrid)
  • -zumab: Humanized (mostly human)
  • -umab: Fully human
  • Example: Infliximab (chimeric), adalimumab (human)

Mechanisms

  • • Target cell surface receptors or circulating proteins
  • • Antibody-dependent cell cytotoxicity (ADCC)
  • • Complement-dependent cytotoxicity (CDC)
  • • Receptor blockade or agonism

Types of Biologics

Therapeutic Antibodies

Cancer Therapy

  • • Rituximab (anti-CD20): Lymphomas
  • • Trastuzumab (anti-HER2): Breast cancer
  • • Bevacizumab (anti-VEGF): Angiogenesis inhibitor
  • • Pembrolizumab (anti-PD-1): Checkpoint inhibitor

Autoimmune/Inflammatory

  • • Infliximab (anti-TNF-α): RA, IBD
  • • Adalimumab (anti-TNF-α): Multiple indications
  • • Ustekinumab (anti-IL-12/23): Psoriasis
  • • Secukinumab (anti-IL-17): Psoriatic arthritis

Vaccines

Live-Attenuated

  • • MMR, varicella, rotavirus
  • • Strong immunity
  • • Contraindicated in immunocompromised

Inactivated/Subunit

  • • Influenza, hepatitis A/B, HPV
  • • Safer in immunocompromised
  • • May need boosters

mRNA Vaccines

  • • COVID-19 (Pfizer, Moderna)
  • • Encode antigen protein
  • • Novel platform

Gene Therapy

Ex Vivo

  • • CAR-T cell therapy (tisagenlecleucel)
  • • Cells modified outside body, then reinfused
  • • For hematologic malignancies

In Vivo

  • • Onasemnogene (Zolgensma) for SMA
  • • AAV vector delivers gene directly
  • • One-time treatment

Biosimilars

Regulatory & Clinical Considerations

Definition:

Biosimilars are highly similar to an approved reference biologic, with no clinically meaningful differences in safety, purity, or potency. NOT identical (unlike generic small molecules).

Approval Process:

  • • Analytical studies (structure, function)
  • • Pharmacokinetic/pharmacodynamic studies
  • • Limited clinical trials (demonstrate similarity)
  • • Less extensive than original biologic approval

Examples:

  • • Filgrastim-sndz (biosimilar of Neupogen/filgrastim)
  • • Infliximab-dyyb (biosimilar of Remicade)
  • • Bevacizumab-awwb (biosimilar of Avastin)

Benefits:

  • ✓ Lower cost (15-35% reduction)
  • ✓ Increased access to biologics
  • ✓ Comparable efficacy and safety

Special Considerations for Biologics

Immunogenicity

  • • Formation of anti-drug antibodies (ADAs)
  • • Can reduce efficacy or cause hypersensitivity
  • • Higher risk with chimeric vs humanized antibodies
  • • May require switching to different biologic

Administration & Storage

  • • Most require refrigeration (2-8°C)
  • • IV infusion or subcutaneous injection
  • • Cannot be taken orally (degraded in GI tract)
  • • Often require premedication (antihistamines, steroids)

Infection Risk

  • • Immunosuppression increases infection risk
  • • Screen for latent TB before TNF-α inhibitors
  • • Avoid live vaccines during therapy
  • • Monitor for opportunistic infections

Cost Considerations

  • • Extremely expensive ($20,000-$500,000/year)
  • • Biosimilars offer cost savings
  • • May require prior authorization
  • • Patient assistance programs often available