The road to biosimilars in rare diseases ‐ ongoing lessons from Gaucher disease

Drelichman, Guillermo; Castañeda‐Hernández, Gilberto; Ar, Muhlis Cem; Dragosky, Marta; Garcia, Ricardo; Lee, Howard; Moiseev, Sergey; Naderi, Majid; Rosenbaum, Hanna; Žnidar, Irena; Zuluaga, Andres F.; Freisens, Selena; Mistry, Pramod K.

doi:10.1002/ajh.25701

Cited by 11 publications

(11 citation statements)

References 6 publications

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“…Although Abcertin and Asbroder both share the same INN imiglucerase with Cerezyme, both have been approved in their respective countries (South Korea and Mexico) via regulatory pathways that are not aligned with WHO Similar Biotherapeutic Product guidelines [19][20][21][22][23]. Abcertin was initially approved in South Korea through a national pathway that permitted the submission of phase III data after approval.…”

Section: Introductionmentioning

confidence: 99%

“…Abcertin was initially approved in South Korea through a national pathway that permitted the submission of phase III data after approval. This pathway is distinct from South Korea’s pathway for approval of biosimilars and other orphan drugs [ 23 ]. Asbroder was approved in Mexico as an orphan drug, a regulatory pathway distinct from the biosimilar pathway [ 22 , 23 ].…”

Section: Introductionmentioning

confidence: 99%

“…This pathway is distinct from South Korea’s pathway for approval of biosimilars and other orphan drugs [ 23 ]. Asbroder was approved in Mexico as an orphan drug, a regulatory pathway distinct from the biosimilar pathway [ 22 , 23 ]. As such, they should not be regarded as biosimilar and interchangeable with Cerezyme [ 22 , 23 ].…”

Section: Introductionmentioning

confidence: 99%

“…Asbroder was approved in Mexico as an orphan drug, a regulatory pathway distinct from the biosimilar pathway [ 22 , 23 ]. As such, they should not be regarded as biosimilar and interchangeable with Cerezyme [ 22 , 23 ].…”

Section: Introductionmentioning

confidence: 99%

“…The shared use of the INN imiglucerase has the potential to cause confusion and misconceptions among regulators, healthcare providers (HCPs; including prescribers, pharmacists, and those who administer drugs), and patients regarding the interchangeability of Cerezyme and other products that use the same INN and have not been approved as biosimilars according to WHO biosimilarity guidelines [ 22 , 30 ]. The use of the same INN imiglucerase may compromise the accuracy of adverse event (AE) data because the reporting of AEs using the INN imiglucerase could result in: (1) forced pooling of AEs for ISU Abxis-derived products and Cerezyme; (2) the opportunity to miss a potential safety signal from one of the drugs; and/or (3) inaccurate attribution of a potential safety signal to the wrong drug.…”

Section: Introductionmentioning

confidence: 99%

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Use of Identical INN “Imiglucerase” for Different Drug Products: Impact Analysis of Adverse Events in a Proprietary Global Safety Database

et al. 2022

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Introduction Approved in 1994 and assigned the International Nonproprietary Name (INN) imiglucerase by the World Health Organization, Cerezyme ® (Sanofi Genzyme) is an enzyme replacement therapy used to treat Gaucher disease in > 90 countries. At least two therapies approved outside the USA and the European Union, Abcertin ® and Asbroder ® , have adopted the identical INN imiglucerase. Both drugs were approved via regulatory pathways not aligned with World Health Organization Similar Biotherapeutic Product guidelines. Objective We analyzed whether the use of the identical INN "imiglucerase" for these drugs impacts adverse event (AE) reporting in the Sanofi Global Safety Database. Methods First, we reviewed all imiglucerase individual case safety reports (referred to as cases) including AE data reported between January 2012 and March 2018 that contained Abcertin or Asbroder in the narrative. In a second analysis, we examined cases from Mexico reported between May 2013 and March 2018 to assess changes in imiglucerase reporting following the 2015 approval of Asbroder in Mexico. Results Fifty-six cases mentioning Asbroder and none mentioning Abcertin were retrieved in the first analysis. Upon close review, the AEs of 45 cases (80.4%) were attributed to Asbroder, one (1.8%) to Cerezyme; the specific drug attribution for the AEs of ten cases (17.9%) could not be determined. In the second analysis, a substantial increase in cases and AEs was observed in the period after Asbroder approval (73 cases with 150 AEs pre-approval vs 132 cases with 333 AEs postapproval). Twenty-three of 132 (17.4%) post-approval cases reported discontinuation of treatment (19 related to Asbroder AEs, and four related to Cerezyme AEs). Infusion-associated reactions occurred in 25/132 cases (17 Asbroder related, six Cerezyme related, two indeterminate). Conclusions This analysis demonstrates two potential consequences of identical INN use between Cerezyme and Asbroder:(1) an aggregate safety profile for Cerezyme that includes other products using the identical INN leading to inaccurate pharmacovigilance data and (2) healthcare providers switching, substituting, or potentially assuming interchangeability between the products. Identical INN use without the brand name differentiator may compromise pharmacovigilance data, potentially masking differences in safety profiles between products.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Use of Identical INN “Imiglucerase” for Different Drug Products: Impact Analysis of Adverse Events in a Proprietary Global Safety Database

et al. 2022

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Recent Therapeutic Advancements for Gaucher Disease

Pradhan,

Manna,

Pragya

et al. 2024

Advanced Therapeutics

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Gaucher Disease (GD) is a well‐known lysosomal storage disease resulting from mutations in the GBA1 gene. GD exhibits a range of clinical manifestations, each with unique symptoms and severity levels. This review explores the genetic foundations of GD, highlighting the significance of Glucocerebrosidase (GCase) deficiency, resulting in the skeletal complications associated with GD, such as osteonecrosis, fractures, and bone pain, all of which significantly negatively influence the quality of life for patients. Over 700 mutations in the GBA1 gene are found to cause variations in gene expression in GD, indicating the disease's complexity and the need for continued research. Early diagnosis and prognosis evaluation depend heavily on diagnostic approaches integrating laboratory assessments, genetic testing, and clinical symptoms. Treatment strategies like enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) have advanced, but issues like high costs and invasiveness still exist. This review focuses on novel therapeutic approaches that show promise in treating GD, including gene and cell‐based therapies, pharmacological chaperone therapy (PCT), and drug delivery via nanoparticles. Finally, discussions on current clinical trials, limitations of the advanced therapies, and future scope are summarized.

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