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Aegerion and NPS Pharmaceuticals begin rare disease product launches

Authored by Karl Simpson

Commercialising a new drug is the privilege of the very few. In recent times, the number of marketing approvals has begun to rise and many of these have carried orphan designations. We look at two companies in this fortunate position, Aegerion Pharmaceuticals and NPS Pharmaceuticals.

Unquestionably, rare diseases and those companies developing therapeutic drugs in this market have been gaining a lot of attention. There is a lot of interest in this segment of the pharmaceutical sector and the potential for strong commercial opportunities due the price many rare disease drugs have been able to achieve.

The world of drug development is fraught with risk. Developing drugs in the rare disease area is no different and is subject to the same levels of risk of those more common diseases. When you have identified a disease target for your molecule, you have conducted the challenging business of clinical trials within a limited patient population, you still have many challenges that lie ahead of you. Ultimately, overcoming the hurdles on the pathway to new therapies means you hopefully get regulatory approval for a marketing authorisation, you’ve sussed out the manufacturing challenges, you have conducted your pricing analysis and you are taking head on the very considerable pricing and reimbursement issues which pervade almost every major healthcare system across the world.

Well, Aegerion and NPS Pharmaceuticals are two companies which in recent times have jumped these hurdles and are running towards the tape of commercialisation. For Aegerion it is JUXTAPID™ and NPS Pharmaceuticals it is GATTEX™. These companies recently announced their quarterly results which transcribe the early indications from these early product launches.

With Aegerion, the early signs of commercial launch seem to be working out well. The company has communicated that they’re seeing higher that initially anticipated interest from cardiologists eager to treat their patients. The company has reaffirmed their estimates that there are 3000 patients in the US with clinical or laboratory diagnosis in system HoFH. At the recent earnings call, Aegerion announced that they now have over 185 global prescriptions from JUXTAPID written, a number which includes patients in the US and commercially-reimbursed named-patients in the certain countries outside the US. This number does not necessary imply that every prescription written is a patient on therapy. That number sat at 75.

This early success in getting JUXTAPID commercialised in the US has yet to deliver sizeable revenues which would provide any indication of significance, however, the forecast for 2013 is for revenues from $15m-$25m. In late May 2013, Aegerion received CHMP positive opinion by the EMA clearing the way for marketing authorisation of LOJUXTA™ in Europe for HoFH. This opens up the commercial opportunities for the product in Europe.

NPS Pharmaceuticals meanwhile is busily launching its Gattex product for Short Bowel Syndrome, another rare disease. At their Q1 2013 earnings call, NPS management said by May 3rd 2013, they’d received 160 Gattex prescriptions of which 42 patients are already on therapy. The company remains highly confident of achieving their 2013 target of between 200-300 patients on Gattex. All of this early launch success generated sales of $654,000 of Gattex net sales for Q1 2013.

The Gattex product, to be marketed in Europe as Revestive™, was originally licensed to Takeda along with Preotact™. NPS Pharmaceuticals has re-aquired the commercial rights from Takeda through an equity transaction of $50m. The company is now pursuing different strategies for its international operations.

Aegerion was a fascinating story of an asset which originally came from BMS and thankfully Dick Gregg (BMS) and Dan Rader (Univ. Pennsylvania) found a way to advance lomitapide, ultimately ending up with Aegerion’s approval of JUXTAPID.

Both companies want to become major global companies focused on providing therapies for rare diseases and the early signals of their commercial launches, despite much coverage of the prices of these respective drugs, is that they are off to a very good start to build that commercial platform.

Posted in M&A Finance and Funding, Orphan Drugs and Rare Diseases, Pharmaceutical business | Tagged , , , , , , , , , , , , , , , , , , , , | Leave a comment

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