Biologic medicines for eye disorders MCQs With Answer

Introduction: This quiz collection on Biologic medicines for eye disorders is designed for M.Pharm students studying Biological Evaluation of Drug Therapy. It focuses on clinically important biologics used in retinal and other ocular diseases — including monoclonal antibodies, antibody fragments, fusion proteins, aptamers and gene therapies. Questions probe mechanisms of action, intravitreal pharmacokinetics, immunogenicity, adverse effects, formulation and handling, regulatory considerations and modern therapeutic approaches. The aim is to deepen understanding beyond memorization so students can critically evaluate biologic selection, dosing rationale, safety monitoring and laboratory methods relevant to ocular biologics and their development.

Q1. Which of the following best describes the mechanism of action of ranibizumab in neovascular age-related macular degeneration (nAMD)?

• Full-length monoclonal antibody that induces complement-mediated lysis of VEGF-expressing cells
• Small-molecule inhibitor of VEGF receptor tyrosine kinase activity
• Monoclonal antibody fragment that binds VEGF-A and prevents it from activating VEGF receptors
• Soluble decoy receptor that degrades VEGF-A extracellularly

Correct Answer: Monoclonal antibody fragment that binds VEGF-A and prevents it from activating VEGF receptors

Q2. Aflibercept differs from bevacizumab primarily because it is:

• A monoclonal antibody directed only against VEGF-B
• A fusion protein combining VEGF-binding domains of receptors with an IgG Fc region
• An RNA aptamer that blocks VEGF-A
• A gene therapy vector delivering a VEGF inhibitor gene

Correct Answer: A fusion protein combining VEGF-binding domains of receptors with an IgG Fc region

Q3. Which of the following ocular biologics is an RNA aptamer originally approved for intravitreal use?

• Ranibizumab
• Pegaptanib
• Bevacizumab
• Conbercept

Correct Answer: Pegaptanib

Q4. The principal reason intravitreal biologics can have prolonged intraocular residence time compared with systemic dosing is:

• The presence of a high concentration of proteases in the vitreous that bind biologics
• Restricted clearance due to ocular barriers and limited lymphatic drainage in the vitreous cavity
• Rapid systemic absorption via conjunctival vessels
• Active transport across the blood-retina barrier into systemic circulation

Correct Answer: Restricted clearance due to ocular barriers and limited lymphatic drainage in the vitreous cavity

Q5. Which adverse ocular event has been specifically associated with brolucizumab in post-marketing reports?

• Corneal decompensation
• Retinal vasculitis and retinal vascular occlusion
• Lens subluxation
• Optic neuritis due to demyelination

Correct Answer: Retinal vasculitis and retinal vascular occlusion

Q6. Immunogenicity of intravitreal biologics is a clinical concern because formation of anti-drug antibodies can:

• Only increase systemic exposure without affecting ocular efficacy
• Neutralize drug activity, reduce efficacy and potentially increase intraocular inflammation
• Cause immediate complement activation in the vitreous in all patients
• Convert the biologic into a small-molecule metabolite

Correct Answer: Neutralize drug activity, reduce efficacy and potentially increase intraocular inflammation

Q7. Voretigene neparvovec (Luxturna) is best described as which class of ocular biologic therapy?

• Monoclonal antibody against mutated RPE65 protein
• AAV-based gene therapy delivering a functional RPE65 gene to retinal cells
• An antisense oligonucleotide that silences RPE65 expression
• A synthetic peptide vaccine targeting photoreceptors

Correct Answer: AAV-based gene therapy delivering a functional RPE65 gene to retinal cells

Q8. Which parameter is most relevant when comparing intravitreal half-lives of different anti-VEGF biologics?

• Systemic renal clearance rate
• Vitreous molecular interactions, molecular size and presence of Fc domain
• Patient body weight
• Hepatic microsomal enzyme activity

Correct Answer: Vitreous molecular interactions, molecular size and presence of Fc domain

Q9. When compounding bevacizumab for intravitreal injection, the major quality concern for pharmacies is:

• Maximizing the amount of Fc glycosylation
• Ensuring sterility and preventing endotoxin contamination
• Altering the amino acid sequence to reduce size
• Increasing systemic potency via PEGylation

Correct Answer: Ensuring sterility and preventing endotoxin contamination

Q10. Which analytical method is most appropriate for quantifying biologic potency of an anti-VEGF monoclonal antibody intended for intravitreal use?

• Mass spectrometry only for molecular weight determination
• Cell-based neutralization assay measuring inhibition of VEGF-induced signaling
• Simple pH measurement of the formulation
• Colorimetric assay for total protein concentration without functional testing

Correct Answer: Cell-based neutralization assay measuring inhibition of VEGF-induced signaling

Q11. A biosimilar to ranibizumab must demonstrate which of the following to gain regulatory approval for intravitreal use?

• Identical amino acid sequence plus identical clinical trial data to the reference product
• High similarity in analytical and functional characteristics and no clinically meaningful differences in safety and efficacy
• Only lower cost compared to the originator product
• Different route of administration to show novel benefits

Correct Answer: High similarity in analytical and functional characteristics and no clinically meaningful differences in safety and efficacy

Q12. Systemic adverse events of intravitreal anti-VEGF therapy are a concern because systemic absorption may:

• Completely neutralize the drug in the eye
• Potentially increase the risk of arterial thromboembolic events by inhibiting systemic VEGF
• Raise intraocular pressure directly through systemic mechanisms
• Cause irreversible hepatic necrosis

Correct Answer: Potentially increase the risk of arterial thromboembolic events by inhibiting systemic VEGF

Q13. The primary pharmacodynamic marker used in clinical trials to assess response to intravitreal anti-VEGF therapy is:

• Serum VEGF concentration
• Optical coherence tomography (OCT) measurement of central retinal thickness and visual acuity
• Electrocardiogram changes
• Urinary protein excretion

Correct Answer: Optical coherence tomography (OCT) measurement of central retinal thickness and visual acuity

Q14. Which property of an intravitreal biologic would most likely increase its probability of eliciting local immune complex–mediated inflammation?

• Being a humanized or fully human antibody fragment
• Frequent injections leading to higher cumulative exposure and formation of anti-drug antibodies
• Lack of an Fc region
• Low molecular weight below 5 kDa

Correct Answer: Frequent injections leading to higher cumulative exposure and formation of anti-drug antibodies

Q15. For gene therapies administered by subretinal injection, one major pharmacological evaluation parameter different from intravitreal biologics is:

• Assessment of transient vitreous drug concentrations only
• Evaluation of long-term transgene expression, retinal cell transduction efficiency and durability
• Measuring systemic drug clearance over hours
• Performing only in vitro potency assays without in vivo studies

Correct Answer: Evaluation of long-term transgene expression, retinal cell transduction efficiency and durability

Q16. Which statement about Fc domains in intravitreal biologics is correct?

• The presence of an Fc domain always reduces intraocular half-life
• Fc domains can interact with FcRn and immune cells, affecting systemic exposure and effector functions
• Fc domains are required for VEGF binding
• Removing the Fc domain increases Fc-mediated complement activation

Correct Answer: Fc domains can interact with FcRn and immune cells, affecting systemic exposure and effector functions

Q17. In designing a clinical trial to compare two intravitreal biologics for diabetic macular edema, which endpoint combination would be most informative?

• Time to first fever episode and serum cytokine levels
• Change in best-corrected visual acuity (BCVA) and reduction in central retinal thickness on OCT
• Urine drug levels and hair growth rate
• Number of conjunctival epithelial cells in impression cytology

Correct Answer: Change in best-corrected visual acuity (BCVA) and reduction in central retinal thickness on OCT

Q18. Which storage condition is generally recommended for most commercially supplied intravitreal biologics to maintain stability?

• Frozen at −20°C to ensure long-term stability
• Room temperature with repeated agitation to prevent settling
• Refrigerated at 2–8°C, protected from light and avoiding freezing
• Maintained at 37°C to match physiological temperature

Correct Answer: Refrigerated at 2–8°C, protected from light and avoiding freezing

Q19. Off-label intravitreal use of bevacizumab for ocular neovascular diseases is primarily driven by:

• Its larger molecular weight compared to ranibizumab
• Lower cost and similar anti-VEGF activity compared to approved agents
• Superior intravitreal half-life proven in randomized trials
• Exclusive approval for intraocular injection by all regulators

Correct Answer: Lower cost and similar anti-VEGF activity compared to approved agents

Q20. Which monitoring strategy is most important to detect early ocular safety signals after introducing a new intravitreal biologic in clinical use?

• Routine monthly measurement of serum creatinine
• Active pharmacovigilance including post-marketing surveillance for intraocular inflammation, endophthalmitis and retinal vascular events
• Measuring glycosylation patterns in patient tears
• Annual chest X-ray screening

Correct Answer: Active pharmacovigilance including post-marketing surveillance for intraocular inflammation, endophthalmitis and retinal vascular events

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