ViiV Wins Fast-Track Review At EMA For HIV Therapy
Executive Summary
A number of new products have been submitted for review at the European Medicines Agency. While marketing authorization applications from ViiV Healthcare and BioMarin Pharmaceutical are being fast-tracked, a submission from Incyte is not.
ViiV Healthcare’s HIV drug, fostemsavir, and PTC Therapeutics’ gene therapy for the central nervous system disorder, AADC deficiency, are among 11 new products for which marketing authorization applications (MAAs) have been submitted to the European Medicines Agency for review.
The other newly filed MAAs for potential approval throughout the EU include BioMarin Pharmaceutical’s gene therapy, valoctocogene roxaparvovec, for hemophilia A, Innate Pharma’s Lumoxiti (moxetumomab pasudotox) for treating hairy cell leukemia (HCL), and Incyte's cholangiocarcinoma treatment, pemigatinib. They also cover Aerie Pharmaceuticals’ glaucoma treatment, Roclanda (netarsudil and latanoprost), Verastem’s Copiktra (duvelisib) for treating common types of indolent non-Hodgkin lymphomas, Kyowa Kirin’s Parkinson's disease drug, istradefylline (KW-6002), Paion’s remimazolam for procedural sedation, and two generic products – azathioprine and glucagon.
Nine of the products are recorded in the EMA’s latest list of MAAs under review, which was compiled on 6 January and includes information only for medicines whose applications have been validated at the time the report was compiled. The submissions of the other two treatments were announced by their sponsors after the list was compiled.
Fast-Track Status For ViiV And BioMarin
Two of the new MAAs – for fostemsavir and valoctocogene roxaparvovec – are being reviewed under the EU’s accelerated assessment mechanism, which the EMA reserves for products it deems to be of major interest for public health, particularly from the point of view of therapeutic innovation. Under the fast-track procedure, the time it takes the agency to evaluate a MAA is reduced from 210 days to 150 days (not counting clock stops when applicants have to provide additional information). Incyte had applied for fast-track review for pemigatinib but its request appears to have been rejected. The product is listed by the EMA as being under standard review.
Fostemsavir is an investigational, first-in-class attachment inhibitor for treating HIV in adults who have few treatment options available, said ViiV, which is majority owned by GSK, with Pfizer and Shionogi Limited as shareholders.
The company is seeking approval of fostemsavir, used in combination with other antiretrovirals, for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive antiviral regimen due to resistance, intolerance or safety considerations. “Although these individuals make up a small percentage of the total number of people who live with HIV, their unmet treatment needs are life-threatening and we are committed to addressing them through innovative new medicines like fostemsavir,” noted ViiV CEO Deborah Waterhouse.
In the US, the company submitted a new drug application (NDA) for fostemsavir to the Food and Drug Administration in December 2019. The FDA has also granted the product fast-track status and breakthrough therapy designation. ViiV said it planned to submit regulatory applications for fostemsavir to other global agencies in the coming months. (Also see "Keeping Track: US FDA's Rejection Of First RMAT BLA Blemishes Otherwise Positive Week Of Non-Oncology News" - Pink Sheet, 9 Dec, 2019.)
BioMarin’s gene therapy, valoctocogene roxaparvovec, is for the treatment of adults with severe hemophilia A. According to the company, this is the first marketing application that has been filed for a gene therapy product for any type of hemophilia. Valoctocogene roxaparvovec was accepted in 2017 into the EMA’s PRIME (priority medicines) scheme, which is designed to get drugs for unmet medical needs to patients faster.
In the US, BioMarin says it remains on track to submit a biologics license application for valoctocogene roxaparvovec to the FDA by the end of the year. The FDA has granted the treatment breakthrough therapy designation. It has orphan drug designation in both the US and the EU. (Also see "EU Accelerated Assessment Tracker" - Pink Sheet, 31 Dec, 2019.)
As for the other non-generic products for which MAAs were newly filed:
- PTC’s gene therapy treatment, PTC-AADC, for AADC deficiency is expected to receive an opinion on whether it should be approved by the EMA's Committee for Medicinal Products for Human Use in the second half of 2020, according to the company. AADC deficiency is a rare central nervous system disorder arising from reductions in the enzyme aromatic L-amino acid decarboxylase (AADC) that result from mutations in the dopa decarboxylase (DDC) gene. In its profound forms, the condition causes severe developmental disabilities, the inability to develop any motor strength and control (global muscular hypotonia/dystonia) resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care. Patients with severe forms often die in the first decade of life due to profound motor dysfunction, autonomic abnormalities and secondary complications such as choking, hypoxia and pneumonia.
- Innate’s Lumoxiti is for treating adults with relapsed or refractory HCL who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Lumoxiti was approved in the US in September 2018. If it is approved in the EU, the company says the product would be the first treatment available in the region for relapsed or refractory HCL patients in more than 20 years.
- Incyte’s pemigatinib is for treating adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy. In the US, the FDA accepted the company’s NDA for the product for priority review last November.
- Aerie's Roclanda is currently marketed as Rocklatan in the US for reducing elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. The MAA for Roclanda follows Aerie’s receipt of pan-EU marketing approval for Rhokiinsa (netarsudil ophthalmic solution) 0.02% in November 2019 (Rhokiinsa is marketed as Rhopressa in the US). In the EU, fixed-dose combinations with prostaglandins are frequently prescribed for reducing intraocular pressure associated with glaucoma. The company says that if the Roclanda MAA is approved, the combination of its Rho kinase inhibitor with a prostaglandin would be a first for Europe.
- Verastem’s Copiktra is an oral inhibitor of phosphoinositide 3-kinase for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and relapsed or refractory follicular lymphoma. Copiktra was approved in the US in 2018 for treating relapsed or refractory CLL/SLL after at least two prior therapies.
- Kyowa Kirin’s istradefylline is an adjunctive treatment to levodopa-based regimens in adult patients with Parkinson's disease experiencing "off" time. Istradefylline, an orally administered, selective adenosine A2A receptor antagonist, was approved in Japan under the brand name Nouriast in 2013 for improving the "wearing-off" phenomenon in patients with Parkinson's disease on levodopa-containing preparations. In August 2019, the product was approved in the US (as Nourianz) for use as adjunctive treatment to levodopa/carbidopa in adults with Parkinson's disease experiencing "off" episodes.
- Paion’s remimazolam is an ultra-short-acting intravenous benzodiazepine sedative/anesthetic for procedural sedation. In the US, Paion’s licensee Cosmo Pharmaceuticals submitted an NDA for remimazolam for use in procedural sedation in April 2019 – a PDUFA decision date of 5 April 2020 has been set. Paion’s licensees in Japan (Mundipharma) and China (Yichang Humanwell) have also filed for market approval for remimazolam. The filing in Japan took place in December 2018 and covers general anesthesia, while the Chinese filing was in November 2018 for remimazolam’s use in procedural sedation.
Details of these latest applications will be added to the Pink Sheet's New Filings at the EMA tracker in due course. The latest update is available here.