| THE FACTS: | | | | specific guideline in March 2009 which illustrates in |
| On July 23, 2010 the U.S. Food and Drug | | | | detail which data are expected for a biosimilar LMWH. |
| Administration FDA approved the first generic version | | | | This guideline requires a PK/PD study in healthy |
| of Lovenox (enoxaparin sodium injection), an | | | | volunteers plus a clinical efficacy/safety study in |
| anti-coagulant drug used for multiple indications | | | | patients being relevant for the clinical use of LMWH |
| including prevention of deep vein thrombosis. FDA | | | | such as those undergoing major orthopaedic surgery. |
| approved an abbreviated new drug application | | | | The FDA has acknowledged the fact as well that |
| (ANDA) for Sandoz‘ product which is a generic | | | | enoxaparin is a biologically-derived product while |
| version of Lovenox, approved for Sanofi-Aventis in | | | | commenting on the approval of the first generic |
| 1993. Enoxaparin, the active ingredient of Lovenox, is | | | | enoxaparin since it referred to the fact that the |
| a low molecular weight heparin (LMWH) with | | | | process can be more complex for a natural product. |
| approximately 4500 Da manufactured from heparin | | | | Despite this, FDA considered it justified to base the |
| by alkaline beta-eliminative cleavage of the benzyl | | | | approval of this biologically-derived product on the |
| ester of heparin. Other LMWHs are authorised which | | | | ANDA legal pathway which is nothing else than |
| use different methods of heparin depolymerisation. | | | | approving it with the criteria applied for a common |
| The heparin underlying the manufacture of LMWHs is | | | | chemical generic drug for the evidence of sameness. |
| derived from natural sources, mainly porcine intestine. | | | | FDA has, however, taken into account the nature of |
| The generic was developed by Sandoz and Momenta | | | | enoxaparin specifically in their review by adapting |
| Pharmaceuticals and applied for FDA approval in 2005 | | | | these criteria by the following five criteria: |
| already. Prior to the approval, the FDA also had to | | | | 1. Equivalence of heparin source material and mode of |
| review a citizen petition questioning the approval | | | | depolymerization |
| criteria for generic enoxaparin sodium injection and | | | | 2. Equivalence of physiochemical properties |
| determined in response to it that it had a sound basis | | | | 3. Equivalence in disaccharide building blocks, fragment |
| for the approval. | | | | mapping, and sequence of oligosaccharide species |
| All primary information related to the FDA decision | | | | 4. Equivalence in biological and biochemical assays |
| including the approval letter and the response to the | | | | 5. Equivalence of in vivo pharmacodynamic profile |
| citizen petition can be found here. | | | | |
| On August 17, 2010, a U.S. federal judge hearing is | | | | These five criteria would have been part of a |
| hold in response to Sanovi-Aventis‘ lawsuit filed | | | | European dossier as well. The first three criteria are |
| against the FDA on July 26, 2010. It says that the | | | | used to ensure that the heparin source material, the |
| FDA has exceeded its authority given by the | | | | chemical reaction used in the manufacturing process, |
| mechanism of ANDA approval. | | | | and the structure (the distribution of molecular |
| THE ISSUE: | | | | weight, chemical composition and sequence) of the |
| Enoxaparin remains a biologically derived product | | | | active ingredient, enoxaparin sodium, in the generic |
| despite the semi-synthetic manufacturing steps used | | | | drug product is equivalent to that in the brand name |
| to depolymerize the heparin from which it is | | | | counterpart, Lovenox. The difference lays in the |
| produced, the resulting low molecular weight and the | | | | interpretation of the fourth and fifth criteria. FDA |
| structure which is possible to be well-characterised by | | | | used them to require the applicant to ensure that the |
| analytical methods. Products derived from a biological | | | | generic enoxaparin sodium has the same degree of |
| source are by definition complex, difficult to fully | | | | anticoagulant activity as Lovenox. And based on all |
| characterise by analytical means and the relationship | | | | these five criteria FDA considered that the generic |
| of structure with clinical effect and safety is difficult | | | | enoxaparin sodium has the same active ingredient as |
| to determine by non-clinical investigations only. This is | | | | Lovenox. |
| common understanding. Accordingly, the basis of | | | | In the EU guideline on biosimilar LMWH, the criterion |
| approval of a generic version of a biologically-derived | | | | five is also included, i.e. the requirement to conduct a |
| product needs to take this into account. In the EU, | | | | study in healthy volunteers to investigate the |
| the legal articles determining the pathways for | | | | pharmacokinetic/pharmacodynamic properties. FDA |
| authorisation of a generic version of a product with | | | | did, however, not insist on the conduct of a clinical |
| chemical/synthetic or biological origin are thus | | | | efficacy/safety study in patients reflecting the clinical |
| different – see article 10 of the EU-Directive 2001 | | | | use of enoxaparin as it would have been the case in |
| 83 as published here. | | | | the EU in addition to the five criteria. The study is |
| Furthermore, there is a difference in terminology: | | | | required in the EU due to the heterogeneity of |
| generics are chemical or synthetic drugs whereas the | | | | LMWH and the uncertainty on the relevance of PD |
| generic version of a biologically-derived product is to | | | | markers for the clinical efficacy. |
| be called a biosimilar. The difference is furthermore | | | | By the way, the EU guideline is intended to be utilised |
| supported by the requirements for the scientific data | | | | for all LMWH applied for authorisation as biosimilars, |
| to be submitted. The approach to develop a biosimilar | | | | independently of an eventually demonstrated |
| requires a comprehensive comparability package | | | | high-grade similarity (EMA) or sameness (FDA) of the |
| which includes – most importantly in the context | | | | active ingredient by other means than clinical testing. |
| of this case – also the requirement to conduct | | | | Thus, it does require the same grade of data as the |
| clinical studies comparative in nature with the | | | | FDA did in this case but plus the clinical study in |
| reference product as the control arm. For LMWH, the | | | | patients. |
| European Medicines Agency EMA has adopted a | | | | |