Roche Holding AG RHHBY announced that the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation to the company’s investigational oral medicine risdiplam (RG7916) for the treatment of people with spinal muscular atrophy (SMA). The EMA grants PRIME status to drugs that have a major therapeutic advantage over existing treatments, or benefit patients without treatment options.
We note that Roche is developing risdiplam, an oral SMN2 splicing modifier, as part of a collaboration with the SMA Foundation and PTC Therapeutics PTCT.
The designation is primarily based on results from Part 1 of the pivotal studies FIREFISH and SUNFISH (in children and adults with Type II and III SMA).
FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type I SMA. Part 1 was a dose escalation study in 21 infants, while Part 2 is a pivotal, single-arm study on risdiplam in approximately 40 infants with Type I SMA for 24 months, followed by an open-label extension.
The primary objective of Part 1 was to assess the safety profile of risdiplam in infants and determine the dose for Part 2. The preliminary data from Part 1 of the FIREFISH study showed that infants with Type I SMA are meeting developmental milestones, including sitting without support.
Six out of 14 infants (43%) were able to sit (with or without support), including three (21%) who achieved unassisted stable sitting after eight months of treatment. Additionally, four infants (29%) demonstrated rolling to the side along with seven (50%) kicking and six (43%) achieving upright head control.
SUNFISH is a two-part, double-blind, placebo-controlled pivotal clinical trial in children and young adults (two to 25 years old) with Type II and III SMA. Part 1 determined the dose for the confirmatory Part 2. Roche completed enrollment in Part 2 in September 2018.
The preliminary data from Part 1 of the SUNFISH study showed improvements in motor function in patients with Type II/III SMA. The follow up studies are ongoing for the confirmatory Part 2 portions of both the studies.
Roche is also conducting a third study, JEWELFISH, which is an open-label exploratory trial in people aged 12-60 years with Type II or III SMA who have been previously treated with SMN-targeting therapy as part of a clinical study.
The company also plans to initiate a new trial, RAINBOWFISH in pre-symptomatic SMA, by early 2019.
Notably, Biogen’s BIIB Spinraza is already approved to treat children and adults with SMA, which reflects the prevailing competition in the market.
Roche has a strong presence in the oncology market. In particular, the company dominates the breast cancer space with strong demand for its HER2 franchise drugs, which include Herceptin, Perjeta and Kadcyla.
The company is making efforts to develop its portfolio beyond oncology, into immunology and neuroscience. Approval of new drugs and diagnostics tests, and label expansion of existing drugs bode well for Roche, given its current challenges.
Year to date, shares of Roche have increased 1.6%, compared with the industry’s growth of 8.4%. Roche’s legacy drugs like Herceptin and MabThera are facing competition from biosimilars. Novartis NVS has already launched its biosimilar version of Rituxan/ MabThera in Europe. Amgen too has got its biosimilar of Avastin.
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Roche currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
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