Biologic medicines for neurological disorders MCQs With Answer

Introduction

This quiz set on Biologic medicines for neurological disorders is designed for M.Pharm students preparing for advanced coursework and competitive exams in Biological Evaluation of Drug Therapy. It focuses on mechanisms, clinical applications, pharmacokinetics, immunogenicity, delivery strategies, regulatory considerations and safety monitoring of biologics used in neurology, including monoclonal antibodies, recombinant proteins, antisense oligonucleotides, cell and gene therapies. Questions are crafted to test applied knowledge, critical interpretation of assays and therapeutic decision-making rather than rote memorization. Detailed answer choices help students understand concepts such as blood–brain barrier strategies, target-mediated drug disposition, and biomarkers relevant to neurologic outcomes.

Q1. Which of the following biologic therapies is a monoclonal antibody used to deplete CD20+ B cells in multiple sclerosis?

• Natalizumab
• Ocrelizumab
• Fingolimod
• Alemtuzumab

Correct Answer: Ocrelizumab

Q2. Antisense oligonucleotide therapy for spinal muscular atrophy that modifies SMN2 splicing is called:

• Nusinersen
• Onasemnogene abeparvovec
• Risdiplam
• Eteplirsen

Correct Answer: Nusinersen

Q3. Which mechanism best describes target-mediated drug disposition (TMDD) commonly seen with many biologics?

• Nonlinear clearance due to saturable binding to a pharmacologic target
• First-order renal excretion unaffected by dose
• Passive diffusion across lipid membranes causing linear kinetics
• Enzymatic metabolism by CYP450 causing dose proportionality

Correct Answer: Nonlinear clearance due to saturable binding to a pharmacologic target

Q4. Which assay is most appropriate for quantifying anti-drug binding antibodies in serum as part of immunogenicity assessment?

• Western blot detecting serum proteins
• Bridging ELISA using drug as capture and detection reagent
• LC-MS/MS quantification of intact biologic
• Flow cytometry of peripheral blood mononuclear cells

Correct Answer: Bridging ELISA using drug as capture and detection reagent

Q5. Progressive multifocal leukoencephalopathy (PML) is an important risk with which monoclonal antibody used in MS?

• Ocrelizumab
• Natalizumab
• Rituximab
• Interferon beta

Correct Answer: Natalizumab

Q6. Which delivery route is most commonly used for antisense oligonucleotides targeting the CNS to bypass the blood–brain barrier?

• Intramuscular injection
• Oral administration with lipid carriers
• Intrathecal administration into cerebrospinal fluid
• Subcutaneous injection with adjuvant

Correct Answer: Intrathecal administration into cerebrospinal fluid

Q7. For AAV-based gene therapy in a neurodegenerative disorder, a major immunogenicity concern is:

• Formation of neutralizing antibodies against the therapeutic transgene product only
• Pre-existing neutralizing antibodies to the AAV capsid that block transduction
• Autoantibodies to myelin basic protein triggered by vector DNA
• Excessive complement activation due to polysorbate

Correct Answer: Pre-existing neutralizing antibodies to the AAV capsid that block transduction

Q8. Which biomarker is increasingly used as a fluid marker of neuronal injury and to monitor response to biologic therapy in multiple sclerosis?

• Creatine kinase
• Neurofilament light chain (NfL)
• C-reactive protein (CRP)
• Beta-amyloid 42

Correct Answer: Neurofilament light chain (NfL)

Q9. A monoclonal antibody engineered to reduce Fc receptor binding would most likely decrease which effect?

• Neutralization of soluble ligand
• Complement-dependent cytotoxicity and antibody-dependent cell-mediated cytotoxicity
• Antigen binding affinity to target epitope
• Transcytosis across neonate Fc receptor into CNS

Correct Answer: Complement-dependent cytotoxicity and antibody-dependent cell-mediated cytotoxicity

Q10. Which pharmacokinetic characteristic distinguishes large protein biologics from small-molecule CNS drugs?

• High oral bioavailability due to size
• Limited passive diffusion across blood–brain barrier and predominant parenteral administration
• Extensive hepatic CYP-mediated metabolism
• Rapid renal clearance as small polar molecules

Correct Answer: Limited passive diffusion across blood–brain barrier and predominant parenteral administration

Q11. What is the primary regulatory submission for approval of a new biologic medicine in the US?

• New Drug Application (NDA)
• Biologics License Application (BLA)
• Investigational New Drug (IND) Application
• Abbreviated New Drug Application (ANDA)

Correct Answer: Biologics License Application (BLA)

Q12. Which of the following best describes a biosimilar for a neurologic biologic agent?

• An identical copy of the reference biologic produced by the same manufacturer
• A product that is highly similar to the reference product with no clinically meaningful differences in safety and efficacy
• A generic small-molecule version with the same active ingredient
• A different molecule with similar therapeutic indication but unrelated structure

Correct Answer: A product that is highly similar to the reference product with no clinically meaningful differences in safety and efficacy

Q13. Which safety monitoring approach is most appropriate post-approval for rare but severe immune-mediated adverse events to a biologic used in neurology?

• Only routine clinical visits without additional reporting
• Risk Evaluation and Mitigation Strategy (REMS) with targeted surveillance and registries
• Discontinue pharmacovigilance after one year
• Rely solely on spontaneous patient reports

Correct Answer: Risk Evaluation and Mitigation Strategy (REMS) with targeted surveillance and registries

Q14. Which of the following is a direct neurotoxin biologic used therapeutically for focal spasticity and dystonia?

• Botulinum neurotoxin (onabotulinumtoxinA)
• Tetanus toxoid
• Alpha-synuclein monoclonal antibody
• Interferon gamma

Correct Answer: Botulinum neurotoxin (onabotulinumtoxinA)

Q15. In the context of biologic PK/PD modeling, which parameter is most informative about prolonged target engagement despite low plasma concentrations?

• Volume of distribution
• Binding affinity (Kd) and target occupancy half-life
• Renal clearance rate
• Fraction unbound in plasma

Correct Answer: Binding affinity (Kd) and target occupancy half-life

Q16. Which cell-based assay is preferred to detect neutralizing anti-drug antibodies that block biological activity of a therapeutic antibody?

• Indirect immunofluorescence on tissue sections
• Cell-based functional assay measuring inhibition of target signaling
• Bradford protein assay
• Direct antigen-binding ELISA without functional readout

Correct Answer: Cell-based functional assay measuring inhibition of target signaling

Q17. For intrathecal biologic delivery, a critical pharmacological consideration compared to systemic dosing is:

• Greater first-pass hepatic metabolism
• Direct exposure of CNS tissue with reduced systemic exposure and distinct clearance via CSF turnover
• Improved oral absorption from CSF
• Increased CYP450 induction in neurons

Correct Answer: Direct exposure of CNS tissue with reduced systemic exposure and distinct clearance via CSF turnover

Q18. Which adverse effect is most commonly monitored with B-cell depleting therapies in neurology?

• Hypoglycemia
• Infusion-related reactions and increased risk of infections
• Severe hypertension due to vasoconstriction
• Hepatic enzyme induction leading to drug interactions

Correct Answer: Infusion-related reactions and increased risk of infections

Q19. A pharmacodynamic endpoint commonly used in clinical trials of ALS gene or biologic therapies to assess motor function is:

• Expanded Disability Status Scale (EDSS)
• Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
• Mini-Mental State Examination (MMSE)
• Montreal Cognitive Assessment (MoCA)

Correct Answer: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

Q20. Which manufacturing attribute is critical for neurologic biologics to minimize immunogenic impurities?

• High endotoxin levels to ensure potency
• Stringent host-cell protein removal and consistent glycosylation profiles
• Use of multiple cell lines interchangeably to increase yield
• Intentional inclusion of adjuvant proteins

Correct Answer: Stringent host-cell protein removal and consistent glycosylation profiles

Download