DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY

• Main Authors: MURRAY JAMES M, COX GERALD F, ANDREWS LAURA P, SCHUCHMAN EDWARD H, DESNICK ROBERT J
• Format: Patent
• Language: English; Korean
• Published: 04.12.2023
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

본 발명은 산성 스핑고미엘리나제 결핍(ASMD), 및 특히 니만-피크 질환(NPD)의 비신경병성 증상, 및 특정 양태에서 B형 NPD를 앓는 인간 피험자를 치료하기 위해 산성 스핑고미엘리나제(ASM)를 사용하는 투여량 단계적 증가 효소 대체 요법에 관한 것이다. The invention relates to a recombinant human acid sphingomyelinase (rhASM) for use in a method of treating an acid sphingomyelinase deficiency (ASMD) in a human subject in need thereof, the method comprising administering to the human subject at least one initial dose of 0.025 mg/kg to 0.05 mg/kg of said rhASM; and administering to the human subject an escalating dose regimen at the following sequential doses: (i) 0.1 mg/kg of said rhASM, (ii) 0.3 mg/kg of said rhASM, and (iii) 0.6 mg/kg of said rhASM, wherein each escalating dose is administered at least once before elevating the dose to the next level.