Which treatments have been recently approved by the regulators in the US & EU?
Until June 30th, 2021, the Food and Drug Administration (FDA) in the US and European Medicine Agency in Europe have launched 32 and 22 new drugs, respectively1. This represents a notable increase in the number of approved molecules by the two regulators during the first half of 20202. There are different speculations what the reason for that might be, but one of them is that regulators have adapted to operate amidst the Covid-19 pandemic. The table below shows the eight most promising recently approved treatments by the FDA or European Medicine Agency (EMA) measured by their sales forecast.
The first in the list is Biogen’s drug Aduhelm (aducanumab) indicated for the treatment of Alzheimer’s disease with an annual sales projection of $3.9 billion in 2024. Its accelerated approval by the FDA earlier this year caused strong controversy as experts believe there is insufficient evidence proving that Aduhelm can address or reverse disease progression. Moreover, Aduhelm was approved at a striking annual list price of $56,000 per patient, which strengthened the controversy around this treatment even more. The approval of aducanumab also resulted in a federal investigation of the experts in FDA who had a meeting with Biogen’s executive prior to the drug’s approval. Debates on Aduhelm’s clinical efficacy are still ongoing but the latest conclusion of FDA’s Peripheral and Central Nervous System Advisory Committee is that “the clinical trial data were not adequate on their own to convincingly demonstrate a clinical benefit in reducing clinical decline in patients with Alzheimer disease”2.
Read more: The story of Adhulem: 1 month post launch
2. Evrenzo (roxadustat)
The European Commission gave its approval for Evrenzo in August 2021. Evrenzo is a first-in-class treatment for adult patients with symptomatic anaemia associated with chronic kidney disease (CKD). The treatment is now the first and only orally administered HIF-PH inhibitor available in Europe, which tackles the condition by activating the body’s natural response to reduced oxygen levels in the blood. The drug has been developed by the US-based company FibroGen who have an agreement with the Japanese drugmaker Astellas Pharma for the commercialisation of the drug in Europe and Japan. In the US, marketing rights to Evrenzo are held by AstraZeneca but the drug has recently faced notable regulatory setbacks as the FDA have recently issued a complete response letter citing safety concerns3. Analysts forecast that Evrenzo can generate $2.7 billion in sales in 20264. However, this forecast was contingent upon a successful FDA approval.
3. Bimzelx (bimekizumab)
Bimzelx is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. As of August 2021, the drug has not been approved by the EMA or FDA. However, Bimzelx received positive opinion from the Committee for medicinal Products for Human Use (CHMP) in Europe in June 2021 so its EMA approval is likely to be realised in the next months. Similarly, the FDA plans on deciding on the approval of the drug in October 2021. Analysts expect the annual sales of Bimzelx to reach up to $2.6 billion in 2026 assuming the drug shows superiority data to Cosentyx5.
4. Vaskepa (icosapent ethyl)
Vaskepa (sold as Vazkepa in Europe) is a medicine that reduces the risk of cardiovascular events such as heart attack, stroke and other problems caused by blocked blood circulation. The drug’s projected peak sales potential in Europe is estimated at $2.4 billion6. However, its developer and manufacturer, Amarin, lost a major patent battle in the Nevada district court against Hikma and Dr Reddy’s, which allowed generic versions of the drug to come to the market. Amarin appealed against the patent decision. The FDA approved Hikma’s generic equivalent to Vaskepa in May 2020. In September 2020, the US Court of Appeals for the Federal Circuit upheld Nevada district court’s decision. That was a commercial catastrophe for Amarin as this officially gave green lights to its generic equivalents in the US. However, it is yet to be seen how the drug will perform in Europe1.
5. Evrysdi (risdiplam)
Commercialised by Genentech in the US and elsewhere by Roche, Evrysdi was approved by FDA in August 2020 for the treatment of spinal muscular atrophy (SMA) in adults, children and infants aged 2 months and older. In April 2021, EMA’s CHMP recommended the approval of Evrysdi for the treatment of 5q SMA in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies and its approval was a fact shortly after that. Evrysdi’s highest annual price in the US is $375,000 per patient and the drug is projected to yield $2.1 billion in sales in 20267.
6. Lumakras (sotorasib)
Amgen’s Lumakras (sotorasib) indicated for treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), who have received at least one prior systemic therapy. This is the first targeted therapy for tumours associated with a KRAS mutation approved by FDA, which represent nearly a quarter of all mutations in NSCLC. The treatment demonstrated tumour shrinkage in around 36% of the patients in clinical trials. Sotorasib will have an annual list price of $214,800 per patient8. The drug’s projected global sales in 2024 are estimated at $1.8 billion.
7. Adtralza (tralokinumab)
Adtralza was approved in June 2021 by the EMA for the treatment of moderate-to-severe atopic dermatitis in adult patients who are candidates for systemic therapy. Additional regulatory filings are underway with the US FDA and other health authorities worldwide. The drug has been developed and will be marketed by LEO Pharma, one of the strongest players in the field of dermatology. Adtralza’s approval was based on efficacy and safety results from the ECZTRA 1,2 and 3 pivotal Phase 3 trials, which included more than 1,900 adult patients with moderate-to-severe dermatitis9. The drug demonstrated superiority over placebo during 16 weeks of treatment across multiple outcome measures reflecting the signs and symptoms of atopic dermatitis10. Adtralza is projected to generate $1.6 billion in sales by 202711.
8. Kesimpta (ofatumumab)
Kesimpta is the first and only self-administered, disease-modifying targeted B-cell therapy for adult patients with relapsing multiple sclerosis, which has been developed by Novartis. The treatment obtained a regulatory approval in the US in August 2020 and in Europe in March 2021. As of August 2021, regulators in Canada, Switzerland, Singapore, Australia and Japan have also given green lights to Novartis to commercialise their treatment in these markets. Approvals were based on two Phase III ASCLEPIOS studies that met primary endpoints where Kesimpta showed a reduction of annual relapses by over 50% versus teriflunomide, a first-line treatment in MS, and achieved more than 30% relative risk reduction of 3-month confirmed disability progression. The treatment may halt new disease activity in patients with relapsing forms of multiple sclerosis, as demonstrated in post hoc analysis, where nearly nine out of 10 patients treated with Kesimpta achieved no evidence of disease activity (NEDA-3) in their second year of treatment12. Kesimpta’s sales projection are estimated at $1.3 billion in its peak sales13.
There is a notable number of treatments, which have the potential to become blockbuster drugs in the upcoming years. Of course, for some of these treatments the sales forecasts are based upon numerous challenges and assumptions, including pending regulatory approvals, post-hoc data collection etc. Moreover, obtaining a marketing authorisation does not guarantee sufficient uptake and commercial success for any drug. To achieve a maximal uptake at their desired price point, manufacturers have to put their products through health technology assessments (HTAs) with healthcare and reimbursement authorities in each country. This is becoming an increasingly challenging endeavour for all pharmaceutical and biotech companies due to the constantly increasing healthcare expenditure resulting in limited payer willingness to fund expensive therapies. That is why, innovative payment methods, such as payment-by-result or managed entry agreements are implemented for these drugs with the aim to minimise uncertainty and make the funding of innovative treatments commercially viable to both parties.
- Wollenberg A, Blauvelt A, Guttman‐Yassky E, Worm M, Lynde C, Lacour JP, Spelman L, Katoh N, Saeki H, Poulin Y, Lesiak A. Tralokinumab for moderate‐to‐severe atopic dermatitis: results from two 52‐week, randomized, double‐blind, multicentre, placebo‐controlled phase III trials (ECZTRA 1 and ECZTRA 2). British Journal of Dermatology. 2021 Mar;184(3):437-49.