Förra veckan meddelade Saniona att amerikanska FDA har beviljat särläkemedelsklassificiering till Tesomet för behandling av hypotalamisk fetma (HO), vilket gör Saniona till det första bolaget som lyckats uppnå denna milstolpe. BioStock kontaktade Rudolf Baumgartner, MD, Chief Medical Officer och Head of Clinical Development på Saniona, för att veta mer om betydelsen av FDAs beslut, samt planerna på att inleda fas IIb-studier under andra halvåret 2021 i både HO och Prader-Willis syndrom (PWS) – en indikation som Saniona fick särläkemedelsklassificiering för från FDA tidigare i år.

Sanionas primära läkemedelskandidat Tesomet – en fastdoskombination av den trefaldiga monoaminåterupptagshämmaren tesofensine och betablockeraren metoprolol – utvecklas för behandling av de sällsynta sjukdomarna hypotalamisk fetma (HO) och Prader-Willis syndrom (PWS). Förutsatt ett positivt resultat i fas IIb-studierna kommer fas III-studier att genomföras innan en ansökan om godkännande av ett nytt läkemedel kan lämnas in i USA och i Europa.

Stärkt case i både HO och PWS

HO kännetecknas av en allvarlig och funktionsnedsättande fetma som ofta återföljs av depression, impulskontrollstörningar, samt en ökad risk för hjärt-kärlsjukdom och typ 2-diabetes.

Det finns idag inga godkända behandlingar för HO, vilket sätter patienterna i en svår position där de hänvisas tillineffektiva alternativ för viktminskning såsom kirurgi, medicinering och livsstilsrådgivning.

I början av mars i år erhöll Saniona särläkemedelsklassificiering för Tesomet för behandling av PWS. Förra veckankunde Saniona alltså säkra sin andra särläkemedelsbeteckning, den här gången rörande Tesomet för behandling avHO. Dessa två viktiga tillkännagivanden medför vissa fördelar för Saniona, såsom fördelaktiga skatte- och potentiella marknadsexklusivitetsfördelar för Saniona och adderar mervärde till dessa kliniska program, vilket understryks av aktiereaktionen och positiva analytiker rapporter efter dessa nyheter.

Sanionas CMO kommenterar

BioStock kontaktade Rudolf Baumgartner, MD, Chief Medical Officer och Head of Clinical Development på Saniona, som ger en mer omfattande bild av FDAs beslut. 

Rudolf, can you briefly talk about HO and why you think no other company has managed to obtain orphan drug status in this indication before?

– HO is a rare disorder caused by injury to a region of the brain known as the hypothalamus. The condition is characterized by rapid, excessive weight gain that persists despite restricted food intake, extreme hunger and several other complications that may include memory impairment, attention deficit, lethargy and other symptoms. HO is rare – it is estimated to impact between 10,000 and 25,000 in the U.S. and between 16,000 and 40,000 in Europe. There has not been a lot of pharmaceutical company research or drug development done for HO, likely because it is rare and it is a complex disorder. This has put Saniona in the position of being a pioneer of drug development in HO and being the first company granted orphan drug designation for this condition.

What does the orphan drug classification mean for Saniona going forward?

– Orphan drug designation qualifies Saniona for certain development benefits, including tax credits, elimination of certain FDA license application fees, and seven years of market exclusivity in the U.S. following approval. Additionally, it allows Saniona to work with and get additional advice and support from the FDA’s reviewing division during our clinical development process. Having this designation for both of our Tesomet programs in PWS and HO is a significant benefit and will allow us to advance Tesomet as quickly and efficiently as possible.

Can you talk about the process that got Saniona to this stage in HO?

– The process for obtaining an orphan drug designation at the FDA is well-established, but it is never a guarantee, and it requires sufficient scientific evidence on both the product and the indication. This is the process that Saniona followed both in PWS and HO. In PWS, where several other biotechs have applied for and received this designation, approval came quickly, back in March. In HO, because the FDA had never granted an orphan drug designation for HO before, the process required a bit more time as the Office of Orphan Drug Products had additional questions on this condition. We were glad to have the opportunity to provide them with the needed information about HO, and were pleased with how quickly they responded and granted this designation in HO.

What does the announcement mean to the HO advocacy community and caregivers?

– We were fortunate to have Amy Wood, Executive Director of the Raymond A. Wood Foundation and parent of a child living with hypothalamic obesity, provide her perspective in our press release. She said: “The recognition of the first orphan drug designation in hypothalamic obesity is a critical milestone for the HO community. … We are incredibly grateful that both the FDA and Saniona recognize the seriousness of this disorder, and we hope this orphan drug designation is the first step towards having an innovative treatment.”

Can you talk about the work that remains before clinical studies can be initiated in the second part of 2021?

– There is a lot of preparation that must be completed to initiate a clinical trial. We have taken many preparatory steps including selecting the clinical research organization (CRO), the manufacturer, and many clinical trial sites for the PWS and HO clinical trials. We are working to complete the FDA’s previous requests for information regarding our manufacturing of Tesomet capsules, and we are on track to start both the PWS and HO phase 2b trials in the second half of this year as targeted.

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